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10/07/2018

Paris, France, July 10th. 2018. Sofinnova Partners, a leading venture capital firm specialized in Life Sciences, today announced that Otsuka Holdings is acquiring its portfolio company ReCor Medical, a medical device company specialized in the treatment of hypertension. The terms of the acquisition are being withheld due to non-disclosure obligations.

ReCor Medical was created in 2009 by Sofinnova Partners, Mano Iyer – who was then entrepreneur-in-residence at Sofinnova Partners and now Chief Operating Officer of ReCor – and Professor Jacques Seguin, MD, who became a large private investor in ReCor. Prof. Seguin was previously founder and CEO of CoreValve, a past Sofinnova portfolio company and a leader in the transcatheter valve replacement space, which was sold to Medtronic. Sofinnova Partners was the sole venture capital investor in ReCor Medical and remained its largest shareholder until the sale to Otsuka.

ReCor Medical is an innovative medical device company that developed the Paradise System, a proprietary ultrasound ablation system for renal denervation (RDN). RDN is a new potential therapeutic approach for the treatment of hypertension, one of the most prevalent medical conditions. ReCor recently announced positive results of its landmark RADIANCE-HTN SOLO hypertension study at EuroPCR 2018.

Antoine Papiernik, Managing Partner at Sofinnova Partners and ReCor Board Member, said: “ReCor perfectly illustrates our investment strategy: we worked hand-in-hand with Mano Iyer to create the business vision and plan for ReCor. We then founded and funded the company, and opened our network of experts, key opinion leaders and board members to help grow it. We brought trusted entrepreneurs Jay Watkins as Chairman and Andy Weiss as CEO to help guide and operate the company through to a corporate transaction to our partner Otsuka.”

Jay Watkins, Chairman of ReCor Medical said: “Sofinnova Partners remains one of few VCs willing to fund early-stage med-tech ventures targeting large and important new markets. The firm played a critical role throughout ReCor’s life, and has proven to be a reliable, value-added partner for the company. The field of renal denervation has been a complex one over the last few years with periods of euphoria and periods of doubt. Sofinnova Partners’ support remained constant throughout, helping to build a strong partnership with Otsuka and then navigate through the challenges to a very successful trade sale.”

Mano Iyer, Founder and COO of ReCor Medical added: “ReCor is a success story because Sofinnova Partners, consistent with its philosophy, saw the value of an opportunity which did not yet exist. It had the vision to create and fund the company, not only in the very beginning, but also during the critical early years. Despite the dramatic swings in the field, Sofinnova Partners’ confidence in me and in the management team was essential to keep us motivated when others lost hope. This great exit is therefore particularly sweet.”

Andrew M. Weiss, CEO of ReCor Medical adds: “I came to ReCor thanks to Antoine Papiernik’s introduction to the company. With his help, our team developed the partnership with Otsuka and was able to remain focused on value creation. The recent announcement of our positive RADIANCE-HTN SOLO study results and now the merger with Otsuka demonstrate that our teamwork with Sofinnova Partners was successful. We now have an opportunity to transform the treatment of hypertension and benefit millions of potential patients while providing a solid return for our investors. I look forward to continuing to work to make this technology a possible standard of care in hypertension treatment”.

For more information, please contact:
SOFINNOVA PARTNERS
International: Anne Rein
Tel: +33 6 03 35 92 05
e-mail: anne.rein@strategiesimage.com
United States: Kate Barrette
Tel: +1 212 223 0561
e-mail: kbarrette@rooneyco.com

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, the firm brings together a team of professionals from all over Europe, the US and China. The firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a lead or cornerstone investor in seed, start-ups, corporate spin-offs and late stage companies. It has backed nearly 500 companies over more than 45 years, creating market leaders around the globe. Today, Sofinnova Partners has over €1.9 billion under management. For more information: www.sofinnova.fr

About ReCor Medical, Inc.
ReCor Medical is a medical device company that designs and manufactures the Paradise System, a proprietary ultrasound ablation system for renal denervation (RDN). RDN is a new potential therapeutic approach for the treatment of hypertension, one of the most prevalent medical conditions. The Paradise System is approved for sale in the EU and bears a CE mark, but is not approved for sale in the United States. The System’s intravascular catheters denervate renal nerves by combining the protection of water-based cooling of the renal artery with high intensity ultrasound energy for circumferential renal nerve ablation. The Paradise System has been studied in clinical trials of approximately 300 patients to date. Following the positive outcomes of the RADIANCE-HTN SOLO trial, ReCor will continue its evaluations of Paradise in RADIANCE-HTN TRIO (a feasibility study of patients with resistant hypertension) and REQUIRE (a pivotal study of patients with resistant hypertension in Japan and Korea), and launch the RADIANCE II pivotal study (a study of patients with moderate hypertension) in the United States and Europe.
http://www.recormedical.com/

About Otsuka Holdings Co., Ltd. and Otsuka Medical Devices Co., Ltd.
Otsuka Holdings Co., Ltd. is the holding company of the Otsuka group, a global healthcare group headquartered in Tokyo, Japan. With operations in pharmaceuticals, nutraceuticals, medical devices and other health-related businesses, the group generated worldwide sales of JPY1,240 billion in the fiscal year ended December 2017.
http://www.otsuka.com/en/

Established in 2011, Otsuka Medical Devices Co., Ltd. is a fully-owned subsidiary of Otsuka Holdings and one of its core operating subsidiaries. Otsuka Medical Devices focuses on the development and commercialization of endovascular devices that provide new therapeutic options in areas where patient needs cannot be met through pharmaceutical or other conventional treatment.
Otsuka Medical Devices conducts the REQUIRE trial for renal denervation in hypertensive patients (n=140), who are uncontrolled on 3 or more medications including a diuretic, in Japan and Korea through its subsidiary JIMRO Co., Ltd.
http://www.omd.otsuka.com/en/

19/09/2017

Key Updates
• ProQR’s drug candidate QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation from the FDA, representing the fifth program in the Company’s pipeline to receive ODD in the U.S.
• The Company will be presenting pre-clinical data for QR-313 at two European scientific conferences in Salzburg, Austria – EB2017 Research Conference and ESDR Meeting.
• DEB is a severe genetic skin disease with no disease modifying treatments currently available.
• QR-313 targets the most common mutations within DEB, which are mutations in exon 73 of the COL7A1 gene and is designed for topical administration.
• A first-in-human clinical trial of QR-313 will be initiated in 2018. Clinical data from the program will also be available in 2018.

LEIDEN, the Netherlands, September 19, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA). QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause in dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for patients.

“We are pleased to have ODD designation in the U.S. for our QR-313 program targeting dystrophic epidermolysis bullosa,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR, “It highlights the unmet need in this devastating disease, for which we aim to make a meaningful difference. Our goal for this disease is to develop a pipeline of programs that can treat DEB mutations in a targeted manner and to actively advance the pipeline through development.”

Poster Presentations at Upcoming Scientific Conferences
The Company will present two posters (# 50 and 51) during the EB2017 – 5th World Conference of Epidermolysis Bullosa Research Conference from September 24-26, 2017 in Salzburg, Austria.
The same posters (# 181 and 194) will also be presented at the 47th Annual European Society for Dermatological Research (ESDR) Meeting on September 29, 2017 in Salzburg, Austria. Poster #181 is selected for a presentation (walk title: Genetics and Cell Based Therapy 2: Epidermolysis bullosa) on September 29 at 14.35-15.30 CET.
The posters are titled:
• Local delivery of an antisense oligonucleotide for recessive dystrophic epidermolysis bullosa.
• In vitro evaluation of QR-313; an antisense oligonucleotide designed to skip exon 73 from the COL7A1 mRNA.
About Orphan Drug Designation (ODD)
Orphan drugs are intended for the treatment, diagnosis or prevention of serious diseases that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. FDA evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. FDA provides incentives for sponsors to develop products for rare diseases, including development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S.

About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. *Since 2012*

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-313 and the clinical development and the therapeutic potential thereof, and statements regarding our pipeline of programs targeting DEB. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that we may not realize the intended benefits afforded by orphan drug designation for our QR-313 program targeting DEB, positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
ProQR Therapeutics N.V.:
Media Contact:
Sariette Witte
T: +31 6 2970 4513 (NL)
T: + 1 213 261 8891 (US)
pr@proqr.com

Investor Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

12/09/2017

Sofinnova Partners announces DNA Script’ successful Series A fundraising of €11 M to advance development of its DNA synthesis technology

Paris, September 12th 2017. Sofinnova Partners, a leading European venture capital firm specialized in Life Sciences, today announced that portfolio company DNA Script, an industry leader in the manufacture of de novo synthetic nucleic acids using proprietary enzymatic technology, has raised €11 million. Sofinnova Partners is DNA Script’s historic and leading shareholder. Alongside existing shareholders which seeded the company, Sofinnova Partners, Kurma Partners, and Idinvest Partners, new high profile investors join the company for this Series A financing round: Illumina Ventures, and Merck Ventures BV (known as M Ventures in the United States and Canada), the corporate venture arm of Merck KGaA.

Founded in 2014 in Paris (France), DNA Script is the world’s leading company in the manufacturing of de novo synthetic nucleic acids using an enzymatic technology. The company aims at accelerating innovation in life sciences and technology through rapid, affordable, and high-quality DNA synthesis. Sixty years after the discovery of DNA, DNA Script has developed a revolutionary approach that leverages nature’s billions of years of evolution in synthesizing DNA to enable genome scale synthesis. The company introduces a novel biochemical process for DNA and RNA synthesis, a fundamental tool used in biology research. DNA Script’s innovation may be used in numerous technologies, such as for instance in the field of electronics for data storage by leveraging unprecedented capabilities of the molecule to store information.

Funds raised will allow DNA Script to strengthen its unique enzymatic technology and nucleotide chemistry platform in order to manufacture high quality oligonucleotides faster, cleaner and more affordably than current market standards.

Joško Bobanović, Partner at Sofinnova Partners, said: “We are excited that DNA Script, which we back since its seed funding, was able to raise such a significant financing. The company is funded by a group of investors who share a collective vision of creating a standalone business that will enable new applications for synthetic DNA and RNA in drug discovery and development, industrial and food technologies”.

Thomas Ybert, PhD, DNA Script Co-founder and CEO, added: “With the success of this financial round, that brings together internationally renowned investors, DNA Script reaches a new stage of its development. It confirms investors’ confidence in the team’s ability to accelerate technical development, identify new markets and business models, and attract interest from major global players”.

This new funding reinforces Sofinnova Partners’ investment strategy in the industrial biotech field initiated in 2009. Being a pioneer investor in this emerging and rapidly growing field, Sofinnova Partners has a current portfolio of ten industrial biotech companies, backed through two dedicated funds: Sofinnova Green Seed Fund which raised €22,5 M in 2012 and Sofinnova IB I which raised €106 M in its first closing in 2017.

Press contact for SOFINNOVA PARTNERS
Anne REIN Tel: +33 6 03 35 92 05 @: anne.rein@strategiesimage.com

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, the firm brings together 12 highly experienced investment professionals from all over Europe, the US and China. The firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a founding and lead investor in start-ups and corporate spin-offs, and has backed nearly 500 companies over more than 40 years, creating market leaders around the globe. Today, Sofinnova Partners has over €1.6 billion under management.

21/06/2017

LE CERCLE/HUMEUR – Développer des licornes est devenu une obsession française, et c’est très bien. Pourtant, lever de l’argent en France reste particulièrement difficile.

Rien ne vaut La Fontaine pour exprimer l’esprit français. Jean de La Fontaine décrit tout au long de ses fables l’homme d’argent comme un être vil, avare, veule. Dans « Le Savetier et le Financier», «Du thésauriseur et du singe» ou dans «L’homme qui court après la fortune et l’homme qui l’attend dans son lit», l’image de ceux qui font de l’argent leur commerce est particulièrement négative. Pas étonnant donc que le financier ait en France une réputation aussi mauvaise, presque pire que celle de l’entrepreneur ou encore du banquier (ou ex-banquier).

Et pourtant, pour les entrepreneurs, les choses semblent en train de changer. Les politiques semblent enfin avoir compris que ce sont eux qui font la France de demain ; que leur volonté d’innover, de prendre des risques pouvait être le porte-étendard de la France qui gagne et regarde devant. L’entrepreneur est en train d’acquérir ses lettres de noblesse et l’ambition de «devenir milliardaire» n’est plus totalement considérée comme un crime.

Un capital-risqueur derrière les licornes

C’est grâce aux start-up innovantes que cette révolution se fait. Criteo, Deezer ou BlaBlaCar dans l’Internet, Cellectis, Corevalve ou DBV dans les sciences de la vie, le succès des entrepreneurs de la tech (digitale ou biotech) explique en grande partie cette évolution des mentalités. Développer des licornes (start-up dotées d’une valeur d’entreprise supérieure à 1 milliard d’euros) est devenu une obsession française, et c’est très bien. Mais il en est tout autre pour le financier, le capital-risqueur derrière chacune de ses entreprises.

Aucune de celles mentionnées plus haut n’aurait pu se développer sans ces apports en capital survenus à des moments risqués de leur vie, pourtant l’image d’Epinal du financier est encore proche de celle décrite par La Fontaine. Le terme même de «capital-risque» décrit bien ce verre considéré à demi vide, à l’opposé du «venture capital» à l’américaine, «capital aventure» qui fait pousser des ailes.

Difficile de lever des fonds

Sofinnova a été créé il y a quarante-cinq ans avec pour seule idée d’être la Société de Financement de l’Innovation. Depuis, elle a financé plus de 500 entreprises en France, en Europe et aux Etats-Unis. Parti avec quelques millions de francs de l’époque, Sofinnova a aujourd’hui sous gestion plus de 1,6 milliard d’euros.

Une minorité d’argent français vient abonder nos fonds.

Une licorne du capital-risque spécialisée des sciences de la vie ? Depuis nos origines, les entreprises du portefeuille ont cumulé plusieurs centaines de milliards de capitalisation boursière. Surtout, les médicaments et produits médicaux que nos fonds ont permis de mettre sur le marché ont soigné des centaines de milliers de patients.

Et pourtant, lever de l’argent en France est particulièrement difficile. A part quelques investisseurs visionnaires, comme bpifrance ou la CNP, une minorité d’argent français vient abonder nos fonds, la majorité venant de souscripteurs européens (suédois, anglais, luxembourgeois, italiens, suisse) ou américains.

Réveiller l’argent qui dort

Ce nouveau gouvernement semble déterminé à investir dans l’innovation. Le fonds de 10 milliards d’euros annoncé par le président Macron la semaine ­dernière à Viva Technology est une excellente nouvelle. Enfin ! Il est aussi important de changer la donne pour les investisseurs en capital-risque, et de promouvoir ce secteur clef pour le financement de l’industrie de demain. Bpifrance fait beaucoup, mais ne peut pas tout faire.

Aux Etats-Unis, ce sont les fonds de pension qui ont financé les fonds qui ont créé Google, Facebook et Amazon. Nous avons un secteur de l’assurance et des banques florissant, mais qui se réfugie souvent derrière les contraintes européennes pour ne pas investir dans les fonds de capital-risque.

Il est urgent de les motiver à le faire. En trouvant les outils pour encourager « l’argent qui dort » à financer ceux qui financent les technologies de rupture, la France pourra faire resurgir des « investisseurs licornes » ayant l’ambition et les moyens pour financer les leaders de l’innovation de demain. Au travail !

Antoine Papiernik est président de Sofinnova Partners
En savoir plus sur https://www.lesechos.fr/idees-debats/cercle/030397629891-sans-financiers-pas-de-licornes-2096060.php#HdSiYVRHvFq2ZGL9.99

20/06/2017
15/06/2017

Montreal, Canada, June 15th, 2017. BioAmber Inc. (NYSE: BIOA, TSX: BIOA) is pleased to announce the launch of BIO-SA™ pharmaceutical grade. This new grade of material provides a USP/NF and FCC Grade of bio-succinic acid manufactured under the United States Food and Drug Administration’s (FDA) good manufacturing practices (GMP) for food and Excipients.  The United States Pharmacopeia (USP), the National Formulary (NF), and the Food Chemicals Codex (FCC) are the public pharmacopeia standards for medicines, food ingredients, dietary supplement products, and ingredients.

These standards are used by regulatory agencies and manufacturers to ensure products are of the appropriate identity, strength, quality, purity and consistency.

“Having met the stringent requirements contained in the NF and FCC reflects BioAmber’s continued dedication to quality through our best-in-class production and purification processes. By achieving this new benchmark, our global customers in the food, pharmaceutical and dietary supplement industries can be assured BioAmber’s facility adheres to the rigorous quality control standards set by the United States Food and Drug Administration (FDA).” said Fabrice Orecchioni, BioAmber’s President & COO. “This grade will allow BioAmber to supply these high-value industries with commercial volumes of an FDA regulated bio-succinic acid, a grade previously unavailable to these markets before today”, he added.

About BioAmber
BioAmber (NYSE: BIOA, TSX: BIOA) is a renewable materials company. Its innovative technology platform combines biotechnology and catalysis to convert renewable feedstock into building block materials that are used in a wide variety of everyday products including plastics, paints, textiles, food additives and personal care products.  For more information visit www.bio-amber.com

Forward-Looking Statements
This press release contains forward-looking statements, which are subject to substantial risks, uncertainties and assumptions. These statements often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may” or similar expressions. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the events and circumstances reflected in the forward-looking statements will be achieved or occur and the timing of events and circumstances and actual results could differ materially from those projected in the forward- looking statements. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.   For additional disclosure regarding these and other risks faced by BioAmber, see disclosures contained in BioAmber’s public filings with the SEC including, the “Risk Factors” section of BioAmber’s most recent Annual Report on Form 10-K and the recent quarterly report on Form 10-Q.

BioAmber Investor Contact
Roy McDowall
Sr. VP Business Development
Tel (514) 844 8000 ext 260
roy.mcdowall@bio-amber.com

15/06/2017

Key program features and updates:

  • ProQR to host an R&D day in New York today, June 15, from 8:00am to 1:00pm Eastern Standard Time. The live webcast can be accessed at www.proqr.com/rd-day.
  • Cystic Fibrosis (CF): Full data from the nasal potential difference (NPD) study for QR-010, ProQR’s lead molecule for CF that is studied in two clinical trials in patients, will be presented, along with an update on the ongoing Phase 1b study, for which topline data are expected in September 2017. Steven M. Rowe, M.D., MSPH, a renowned expert in cystic fibrosis research, will discuss the importance of NPD as a biomarker and the unmet needs remaining in the CF treatment landscape.
  • Leber’s Congenital Amaurosis (LCA): Details of the clinical trial for QR-110, ProQR’s lead molecule in the ophthalmology pipeline. Additional preclinical data will be presented.
  • Ophthalmology pipeline: An update and data for the next four programs in the ophthalmology pipeline will be provided, including: QRX-411 and QRX-421 for Ushers syndrome, QRX-504 for Fuchs Endothelial Corneal Dystrophy (FECD) and QRX-1011 for Stargardt’s Disease. Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness will provide a background on inherited retinal diseases.
  • Dystrophic epidermolysis bullosa (DEB): an update on key pre-clinical functional and delivery data, and an update on preparation for clinical development to be provided for QR-313. M. Peter Marinkovich, M.D., a dermatologist and Director of the Stanford EB disease clinic, will discuss the unmet need in DEB and the current treatment landscape.
  • Axiomer®: ProQR will introduce its novel, proprietary RNA editing platform technology.  Art Levin, Ph.D., an internationally recognized expert on the development of oligonucleotide-based therapeutics, will discuss the evolution of RNA therapeutics.
  • As of March 31, 2017, the Company’s current cash of €52.1 million provides a runway into Q3 2018.

LEIDEN, the Netherlands, June 15, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR), a development stage RNA therapeutics company will provide an update on its product candidates today at an investor event, and introduce Axiomer®, a novel RNA platform technology it pioneered. The R&D Day is hosted by the Company’s management team and will include perspectives from several key opinion leaders. ProQR’s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development.

“Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” said Daniel A. de Boer, CEO of ProQR. “We are pleased to show the progress we have made in our RNA therapeutics pipeline at our second R&D day.  Following QR-010 for CF, QR-110 is now in clinical trials for LCA 10, and our third molecule, QR-313 for DEB will move to clinical trials early next year.  We are focusing on three important genetic diseases, all with high unmet needs, and all which we believe could greatly benefit from our unique RNA oligonucleotide approach. Within the next 18 months we will have generated clinical data in patients in all these programs.”

Axiomer®editing the RNA
ProQR is pioneering a next-generation RNA technology called Axiomer®, which we believe has the potential to yield a new class of medicines for genetic diseases. Axiomer® can make single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G). A member of the Scientific Advisory Board, Dr. Levin will present the landscape and evolution of RNA therapeutics and provide his perspective on this unique and proprietary platform technology.

“While our focus and priority is on clinical development of our most advanced RNA-based therapeutics to help patients with CF, LCA 10 and DEB, we continue to innovate in RNA science. This innovation effort has led to the discovery of a novel RNA editing technology that we believe can address the underlying cause of a broad range of genetic defects at the RNA level,” said Daniel de Boer.  “The invention and patenting of Axiomer® can drive drug discovery and development of a new class of therapeutics, independently and through partnerships.”

The R&D Day will feature presentations by ProQR senior management including Daniel de Boer (Chief Executive Officer), Noreen Henig (Chief Medical Officer), Peter Adamson (Head of Ophthalmology), Gerard Platenburg (Chief Innovation Officer) and David Rodman (Chief Development Strategy Officer).  Discussions will focus on a review and introduction of several near- and medium term value drivers and progress on the Company’s pipeline. In addition, several leading medical researchers will discuss the state of the art in research and development in relation to the company’s pipeline:

  • Steven M. Rowe, M.D., MSPH, Professor, Department of Medicine, Pediatrics, and Cell Developmental & Integrative Biology, and Director Gregory Fleming James Cystic Fibrosis Research Center University of Alabama.

Dr. Rowe is a respected academic physician scientist and a pioneer in the field of personalized therapeutics for cystic fibrosis, cutting-edge discovery in airway disease biology, and translational research in COPD.  He is an international authority in the design and conduct of clinical trials targeting the basic CF defect, and has made key advances in the measurement and interpretation of CFTR function. He directs the Cystic Fibrosis Research Center at UAB, which involves over 100 faculty members and has been continuously funded for over 25 years.  A board-certified physician, Dr. Rowe serves as a Special Consultant for Translational Science for the Cystic Fibrosis Foundation.  He presently has a laboratory of over 25 individuals, embracing lung research from basic discovery, to translational science, to clinical application.

  • Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness.

Dr. Rose oversees the day-to-day operations of the Foundation Fighting Blindness’ Science Department. He also works closely with the clinical arm of the Foundation to establish a seamless pipeline of studies to move preventions and treatments into clinical trials, partnering with biotech and pharma to maximize potential commercialization.  Prior to joining the Foundation in 2004, Dr. Rose was a Director in the NIH Office of Science Policy, where he provided oversight on issues regarding recombinant DNA, including human gene transfer clinical protocols. Dr. Rose currently sits on the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee and is a Health Research Alliance Board member.

  • M. Peter Marinkovich, M.D., Associate Professor, Blistering Disease Clinic Department of Dermatology, Stanford University School of Medicine.

Dr. Marinkovich is an Associate Professor of Dermatology, a faculty member of the Program in Epithelial Biology and the Stanford Cancer Biology Program. He has an interest in inflammatory skin disease and is Director of the Stanford Epidermolysis Bullous Disease and Psoriasis Clinics. He is also an attending dermatologist at the VA Palo Alto Medical Center. Dr. Marinkovich’s research focuses on pathogenesis and therapy of epidermolysis bullosa, psoriasis, hair disorders and skin cancers.

  • Art Levin, Ph.D., international RNA expert and member of the ProQR Scientific Advisory Board.

Dr. Levin has three decades of experience in RNA drug development from discovery through drug registration, both in large pharma and biotech companies. He has been key to the development of numerous of oligonucleotides, including the first approved antisense medicines, and the first microRNA-targeted therapeutic in clinical trials. Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society.  He has served on ProQR’s Scientific Advisory Board since the company’s inception.

R&D Day Event details

Today, ProQR will host an R&D day in New York, NY from 8:00am to 1:00pm ET.  Please email Ronen Abergel, rabergel@troutgroup.com to receive more information and to reserve a seat.

Webcast
The live webcast can be accessed at www.proqr.com/rd-day. The archived webcast of the presentation will be accessible from the ‘Investor Relations’ section of ProQR’s website (www.proqr.com) under ‘Events and Presentations’. The archived webcast will be available for 90 days following the presentation date.

FORWARD-LOOKING STATEMENTS 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the therapeutic potential of our RNA technology, our innovation programs, the timing of our clinical programs and availability of data and our R&D day. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

08/06/2017

Amsterdam, the Netherlands, 8 June 2017, 8.45 am CET – Avantium N.V. proudly announces that a BBI subsidy of EUR 25 million has been granted to the Industry Consortium “PEFerence”. The consortium, consisting of 11 companies including Synvina, Avantium, BASF, a number of reputable industrial companies and iconic brand owners. The partners will jointly work on establishing an innovative supply chain for FDCA and PEF, including the intended construction of a 50,000 tons reference plant in Antwerp.
More details can be found in the below press release from Synvina, Avantium’s joint venture with BASF.
EUR 25 million subsidy granted: Industry Consortium receives funds for polyethylenfuranoate (PEF)
/ European Joint Undertaking on Bio-Based Industries (BBI) awards consortium with 25 million Euro
/ Synvina coordinates industry consortium “PEFerence”
/ Grant for establishing a complete value chain for the use of bio-based furandicarboxylic acid (FDCA) for PEF
/ Synvina intends to build a 50,000 tons plant for FDCA
The European Joint Undertaking on Bio-Based Industries (BBI), consisting of representatives from the European Union and the bio-based industry, granted 25 million Euro to “PEFerence”, a consortium of eleven companies. The grant supports the establishment of an innovative value chain for bio-based raw materials as well as chemicals and materials based on polyethylenefuranoate PEF. It includes the intended construction of a 50,000 tons FDCA reference plant, the main chemical building block for the production of PEF. Synvina will be coordinating the “PEFerence” project.
BBI acknowledges the engagement of “PEFerence” for more eco-friendly materials and end products, resulting in substantial benefits for the environment and society. Based on renewable feedstock, products made of PEF will significantly help to replace fossil-based packaging materials and to reduce greenhouse gas emissions, following BBI’s assessment. PEF is an innovative polyester suitable for applications such as bottles, films and polyurethanes.
PEF: benefits for environment and society
PEF bottles can be recycled and used again as raw material for bottles, as well as for packaging and textiles. Additionally, PEF offers superior product properties in comparison to conventional polyethylene terephthalate (PET) and provides major advantages for end consumers. Improved barrier properties for gases allow to redefine packaging solutions based on PEF. It also offers a higher mechanical strength, thus thinner PEF packaging can be produced and fewer resources are required. PEF is suitable to produce bottles for carbonated and non-carbonated beverages, foil pouches as well as personal and home care products.
Consortium from raw material producer to brand owner
Besides Synvina and its shareholders BASF and Avantium, the partners in the PEFerence consortium are:
· Tereos Participations (France),
· Alpla Werke Alwin Lehner GmbH & Co Kg (Austria), OMV Machinery Srl (Italy) and Croda Nederland B.V. (The Netherlands),
· Nestec Sa (Switzerland) and Lego System As (Denmark),
· Nova-Institut für politische und ökologische Innovation GmbH (Germany) and Spinverse Innovation Management Oy (Finland).
“The grant of the BBI is a strong signal for Synvina and our partners along the value chain to continue our mutual process to make PEF commercially available”, said Patrick Schiffers, CEO of Synvina, and continued: “To open up a market for a new plastic based on renewable feedstock is a major challenge that we best meet with strong partners and our combined expertise. We share the common goal to get PEF commercially to the market thereby providing the market materials with superior properties and to establish sustainable and bio-based plastic value chains.”

About “Public Private Partnership on Bio-Based Industries”
The European Joint Undertaking on Bio-based Industries (BBI) is a public-private partnership between the European Union and the Bio-based Industries Consortium aiming at increasing investment in the development of a sustainable bio-based industry sector in Europe. It aims at providing environmental and socio-economic benefits for European citizens, increasing the competitiveness of Europe and contributing to establishing Europe as a key player in research, demonstration and deployment of advanced bio-based products and biofuels. The BBI Joint Undertaking will also play an important role in achieving a bioeconomy in Europe. Please find further information here.

About Synvina
Synvina is a Joint Venture of Avantium and BASF, located in Amsterdam. Operating a pilot plant in Geleen, the Netherlands, Synvina produces and markets furandicarboxylic acid (FDCA) from renewable resources on pilot plant scale and markets the new polymer polyethylenefuranoate (PEF). Synvina aims to commercialize their activities in the future. FDCA is a building block for various products. Most significantly the polyester PEF, which is suitable for food and beverage packaging and for fibers for carpets and textiles. For the packaging industry, PEF offers superior characteristics like improved barrier properties and a higher mechanical strength enabling thinner packaging. PEF is recyclable. Our strength lies in the combination of our mother companies’ expertise. We merge technology leadership with market leadership. Creativity with reliability. Innovative spirit with production excellence. Together, we aim to become market leaders in FDCA and PEF. Please find further information at www.synvina.com.

08/06/2017

Key Updates
• An oral presentation on the final results from the proof-of-concept (PoC) nasal potential difference (NPD) trial will be given by Steve Rowe, MD at the European Cystic Fibrosis Society (ECFS) conference.
• Preliminary data from the Phase 1b study, PQ-010-001 single ascending dose (SAD) cohorts of the ongoing Phase 1b will be presented in a poster, demonstrating single dose safety and evidence of systemic exposure following administration via inhalation in CF patients.
• Cohort 7 is completed and enrollment of the final cohort in the Phase 1b study, PQ-010-001, is expected to be completed in June 2017.
• Topline safety and exploratory efficacy data from the multiple dose cohorts in the Phase 1b trial are expected to be released in September 2017.

LEIDEN, the Netherlands, June 08, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced presentation of data from two clinical studies of QR-010 in oral and poster sessions at the ECFS conference in Sevilla, Spain from 8 to 10 June 2017. The company also released preliminary data from the ongoing Phase 1b study, demonstrating safety and systemic uptake of QR-010 after a single dose through inhalation.
Oral presentation on June 9
Steve Rowe, M.D., professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and director of the CFF Therapeutics Development Network will give an oral presentation titled “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation [Abstract #WS13.1]”. The presentation will take place on Friday 9 June during the session “New therapies targeting CFTR: what’s new from the clinical trials pipeline?” from 15:00 – 16:30 central European time in Sevilla, Spain.
Poster presentation on June 9
The Company will also present a poster titled: “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation [Poster #40]” during the session “Cell Biology/Physiology/New Therapies” on Friday 9 June 2017 from 14:00 – 15:00 central European time in Sevilla, Spain.
“QR-010 is an innovative approach to restoring CFTR function in patients with CF due to the F508del mutation. Last year, we demonstrated that QR-010 restores CFTR function as measured by a very specific assay, the nasal potential difference. Now we have shown that QR-010 can be detected in the blood following a single dose inhalation. We believe these results support the potential that QR-010 can treat all manifestations of CF,” said Noreen R. Henig, MD, Chief Medical Officer of ProQR. “I am very pleased that enrollment of the Phase 1b study is expected to be completed this month and we are looking forward to unblinding the study and report the top-line data from this phase 1b trial.”

About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

About QR-010
QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. QR-010 has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

About Cystic Fibrosis
Cystic fibrosis (CF) is the most common fatal inherited disease in the Western world and affects an estimated 65,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick, buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 27 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 70% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation.

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-010, our ongoing and planned discovery and development of QR-010 and its therapeutic potential, timing of enrollment and results from our clinical trials, and statements regarding the coverage of our patent portfolio, owned and in-licensed, including the duration of patent coverage. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

08/06/2017

 The completion of the Single Dose Tolerance study (SDT) for CER-209
 No drug related safety nor tolerance issues identified
 The pharmacokinetics observations support CER-209 once daily oral dosing
 Multiple Dose Tolerance study (MDT) now ready to proceed

Toulouse, FRANCE, Ann Arbor, UNITED-STATES, June 7, 2017, 07:00 pm cet – Cerenis Therapeutics (FR0012616852 – CEREN – PEA PME eligible), an international biopharmaceutical company dedicated to the discovery and development of innovative lipid metabolism therapies for treating cardiovascular and metabolic diseases announces today positive results in the SDT Phase I development of CER-209 for the treatment of liver diseases NAFLD (Non-Alcoholic Fatty Liver Disease) and NASH (Non-Alcoholic Steato-Hepatitis).
The objective of the Single Dose Tolerance study carried out in the USA was to assess the safety, tolerability and pharmacokinetics of CER-209 when taken orally as a single dose. Escalating doses of 1, 3, 10, 30 mg were tested on 24 subjects who were treated in four cohorts of 6 subjects. In each cohort, four subjects were treated with active study medication and two subjects with placebo.
“The positive results from the Single Dose Tolerance study allows us to proceed to the next stage of the Phase I clinical development with a Multiple Dose Safety and Tolerance study. Given the current lack of treatment options for NAFLD and NASH, CER-209 has the potential to play an important role in treating hepatic steatosis and atherosclerosis. CER-209’s major asset in NAFLD and NASH treatment lies in its ability to promote HDL recognition and lipid elimination by the liver, through the activation of natural metabolic pathways mediated by the P2Y13 receptor. In addition, the study’s confirmation that the pharmacokinetics of CER-209 permit once daily oral dosing is excellent news for patient treatment”, said Dr. Jean-Louis Dasseux, CEO of Cerenis.
CER-209, an agonist of the P2Y13 receptor, is well suited to the treatment of NAFLD and NASH. NAFLD, a precursor of NASH, is a disorder that is now considered as the most common liver disease in the Western world, impacting 30% of the world’s population1. In addition, epidemiological studies demonstrate that the cardiovascular risk is increased in patients with hepatic steatosis and that the cardiovascular diseases associated are the leading causes of death in patients with liver steatosis1,2.
In preclinical models, CER-209 resulted in a marked reduction in steatohepatitis as determined by reductions in cholesterol, triglycerides and fatty acids in the liver compared with placebo. CER-209 exerts its beneficial effect on liver steatosis via a specific action on the lipid elimination pathways. Furthermore, CER-209 produced considerable decreases in liver enzymes (ALT and AST) in the plasma.

These effects suggest the restoration of liver integrity and indicate a strong potential for CER-209 to treat NAFLD/NASH and to lower the risk of associated cardiovascular disease. CER-209 exerts its beneficial effect on liver steatosis via a specific action on the cholesterol elimination pathways and has robust potential as a treatment for NASH and NAFLD.

About P2Y13 receptor
The P2Y13 receptor is a member of the P2Y receptor family, a well-known “druggable” receptor family including the P2Y12 receptor that is the target of successful drugs such as the anti-thrombotic agent Clopidogrel (Plavix®). P2Y13 deficiency in preclinical models reduces biliary lipid secretions and fecal loss of cholesterol and bile acids.
Deficiency leads to impaired Reverse Lipid Transport from macrophages to feces. P2Y13 receptor activation by small molecule ligands stimulates plasma HDL clearance and HDL endocytosis by the liver so increasing biliary lipid secretion and stimulating overall RLT3.

About CER-209
CER-209 is the first drug candidate in the category of oral P2Y13 receptor agonists. CER-209 is a specific agonist of the P2Y13 receptor and does not interact with the P2Y12 receptor. In preclinical studies CER-209 promotes HDL recognition by the liver and increases Reverse Lipid Transport (RLT), thereby impacting atherosclerosis regression. Because of the favorable metabolic effects observed in the liver, CER 209 may also offer a new mechanism for the treatment of Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steato-Hepatitis (NASH).

About Cerenis: www.cerenis.com
Cerenis Therapeutics is an international biopharmaceutical company dedicated to the discovery and development of innovative lipid metabolism therapies for the treatment of cardiovascular and metabolic diseases. HDL is the primary mediator of the reverse lipid transport, or RLT, the only natural pathway by which excess lipids is removed from arteries and is transported to the liver for elimination from the body.
Cerenis is developing a portfolio of lipid metabolism therapies, including HDL mimetics for patients with genetic HDL deficiency, as well as drugs which increase HDL for patients with a low number of HDL particles to treat atherosclerosis and associated metabolic diseases including Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steato-Hepatitis (NASH).
Cerenis is well positioned to become one of the leaders in the HDL therapeutic market, with a broad portfolio of programs in development.
Contacts:
Cerenis
Jean-Louis Dasseux
CEO
info@cerenis.com
+33 (0)5 62 24 09 49

NewCap
Investors relations
Emmanuel Huynh / Louis-Victor Delouvrier
cerenis@newcap.eu
+33 (0)1 44 71 98 53
NewCap
Media relations
Nicolas Merigeau
cerenis@newcap.eu
+33 (0)1 44 71 94 98

31/05/2017

Key Updates
• ProQR receives Fast Track designation by the U.S. Food and Drug Administration (FDA). Closer interaction with FDA could potentially accelerate the development of QR-110 in patients with Leber’s Congenital Amaurosis Type 10 (LCA 10).
• LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development.
• QR-110 is currently in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, tolerability, pharmacokinetics and efficacy of multiple administrations of QR-110 in one eye of each patient and will include approximately 6 adults and 6 children with LCA 10.
• Top-line trial results are expected in 2018.

LEIDEN, the Netherlands, May 31, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for QR-110, the lead molecule in its ophthalmology pipeline. QR-110 is being developed for the treatment of patients with Leber’s Congenital Amaurosis Type 10 (LCA 10), a rare genetic disease that causes individuals to lose sight, often in the first years of life. QR-110 is a novel investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children and currently there are no disease modifying therapies commercially available or in clinical development.
Fast Track designation is granted by the FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.
“QR-110 is a unique and elegant approach to addressing the underlying genetic defect that leads to blindness in individuals with LCA 10 due to the p.Cys998X mutation. We are very pleased with granting of the Fast Track designation by the FDA for this program as it highlights the need for innovative and efficacious medicines for this devastating disease for which there is currently nothing available,” said Noreen R. Henig, Chief Medical Officer of ProQR. “We are also excited to be able to initiate our first trial for QR-110 as a multidose study and for that we will benefit from the Fast Track Designation. We believe development of QR-110 also opens the possibilities for RNA approaches to target other causes of genetic blindness. We are building our pipeline in ophthalmology and will use our rapid development approach to QR-110 as a model for how to bring RNA therapeutics to patients in need.”

About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

About QR-110
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union.

About Leber’s Congenital Amaurosis Type 10
Leber’s congenital amaurosis is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA Type 10 (LCA 10) is one of the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is most common. LCA 10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA 10 because of this mutation.

About the PQ-110-001 Study
PQ-110-001 is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe.
The primary endpoints will be safety and tolerability. Secondary efficacy endpoints will assess the pharmacokinetics and restoration/improvement of visual function and retinal structure through ophthalmic tests such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in LCA subjects will also be evaluated. Top-line results from the trial are expected to be available in 2018.

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-110 and the clinical development and the therapeutic potential thereof, statements regarding PQ-110-001, including trial design and expected timing of results, statements regarding Fast Track designation, and statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com