usherin

-- On-track to initiate IND-enabling activities for Wilson’s Disease program in 4Q 2024, with IND or CTA filing expected in 1H 2026 -- CAMBRIDGE, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the Company will highlight advances from across its Prime Editing pipeline and platform at the upcoming European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, being held October 22-25, 2024 in Rome, and the American Association for the Study of Liver Diseases (AASLD), being held November 15-19, 2024 in San Diego. “At the ESGCT and AASLD meetings, we will present new preclinical data from across our pipeline, including from our efforts in liver disease,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine. “We are particularly excited to share in vivo data from our Wilson’s Disease program, as well as from our universal liver-directed lipid nanoparticle (LNP) platform. Together, these data reinforce our strategy of prioritizing Wilson’s Disease – a large, genetically defined condition for which there are no currently approved disease-modifying treatments, and which we believe is uniquely suited to a Prime Editing-based approach – and reinforce the potential for our modular universal LNP to be used broadly across current and future programs. We look forward to advancing our Wilson’s Disease program into IND-enabling studies later this year and, longer term, to potentially leveraging the modularity of our platform to expand into numerous follow-on indications.” Details of the presentations are as follows: European Society of Gene and Cell Therapy 31st Annual Congress (ESGCT); October 22-25, 2024 in Rome Presentation Title: LNP delivered Prime Editors restore glycemic control in humanized rodent models of Glycogen Storage Disease Type 1b (GSD1b)Date & Time: October 24, 2024, 2:00 p.m. CEST Poster Title: Development of PM359, a Prime Edited CD34+ cell drug product for the treatment of p47phox Chronic Granulomatous DiseaseDate & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST Poster Title: An all-Prime Editing one-step approach for non-viral generation of a multiplex-edited allogeneic CAR-T cell productDate & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST Poster Title: Methods for genome-wide detection of single strand breaks induced by gene editors reveals the specificity of SpCas9 nuclease domains and provides comprehensive lists of potential off-targets for Prime EditorsDate & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST Poster Title: Exploring the roles of DNA repair processes across diverse Prime Editing strategies with pooled screens and Knock-Knock PrimeDate & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST Poster Title: Prime Editing advancements enable in vivo therapeutic correction of ATP7B p.H1069Q and p.R778L mutations in Wilson’s DiseaseDate & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST Poster Title: Prime Editors precisely correct pathogenic mutations in RHO and USH2A associated Retinitis Pigmentosa and prevent retinal degenerationDate & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST American Association for the Study of Liver Diseases (AASLD); November 15-19, 2024 in San Diego Presentation Title: LNP delivered Prime Editors restore glycemic control in humanized rodent models of Glycogen Storage Disease Type 1b (GSD1b)Date & Time: Sunday, November 17, 2024, 5:00 – 6:30 p.m. PT Presentation Title: Advances in Prime Editing enable in vivo therapeutic correction of the ATP7B p.H1069Q and p.R778L mutations causing Wilson’s DiseaseDate & Time: Monday, November 18, 2024, 8:00 – 9:30 a.m. PT About Prime Medicine Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types. Taken together, Prime Editing’s versatile gene editing capabilities could unlock opportunities across thousands of potential indications. Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: hematology, immunology and oncology, liver and lung. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit www.primemedicine.com. © 2024 Prime Medicine, Inc. All rights reserved. PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are trademarks of Prime Medicine, Inc. All other trademarks referred to herein are the property of their respective owners. Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements about Prime Medicine’s beliefs and expectations regarding: the timing, progress, and results of its Wilson’s Disease program, including the timing of IND-enabling activities and opening an IND and/or CTA application; the initiation, timing, progress, and results of its research and development programs, preclinical studies and future clinical trials; the modularity of the Prime Editing platform and the benefits thereof; the potential for its modular universal LNP to be used broadly across current and future programs; the potential for Prime Editors to more precisely and effectively achieve genetic modification; the potential for Prime Editors to repair genetic mutations and offer curative genetic therapies for a wide spectrum of diseases; the potential of Prime Editors to reproducibly correct disease-causing genetic mutations across different tissues, organs and cell types; its expectations regarding the breadth of Prime Editing technology and the implementation of its strategic plans for its business, programs, and technology; and the potential of Prime Editing to unlock opportunities across thousands of potential indications. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the authorization, initiation, and conduct of preclinical and IND-enabling studies and other development requirements for potential product candidates, including uncertainties related to opening INDs and obtaining regulatory approvals; risks related to the development and optimization of new technologies, the results of preclinical studies, or clinical studies not being predictive of future results in connection with future studies; the scope of protection Prime Medicine is able to establish and maintain for intellectual property rights covering its Prime Editing technology; and the effect of unfavorable macroeconomic conditions or market volatility resulting from general economic, industry and market conditions, including rising interest rates, inflation, and adverse developments affecting the financial services industry. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Prime Medicine’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Prime Medicine’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Prime Medicine explicitly disclaims any obligation to update any forward-looking statements subject to any obligations under applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Investor ContactHannah DeresiewiczPrecision AQ212-362-1200hannah.deresiewicz@precisionaq.com Media ContactDan Budwick, 1ABdan@1ABmedia.com

Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform and continued advancement of pipeline programs, AX-0810 and AX-1412, focused on genetic diseases originating in the liver LEIDEN, Netherlands, CAMBRIDGE, Mass., and CLERMONT FERRAND, France, Aug. 01, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, and Laboratoires Théa, the leading independent eye care group in Europe (“Théa”), today announced an agreement in which ProQR will divest its late stage ophthalmic assets, sepofarsen and ultevursen, to Théa. Under the terms of the agreement, ProQR will receive an initial payment of €12.5M and will also be eligible for up to €135M in further development, regulatory, and commercial payments, as well as additional earn outs up to high teens percentage based on commercial sales in the US and EU. “Théa’s proven expertise in the research, development, and commercialization of eye care products makes them the ideal company to continue the development of sepofarsen and ultevursen for patients with rare genetic eye diseases,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “We look forward to continuing to advance our Axiomer RNA editing platform, with an initial focus on targets for cholestatic and cardiovascular diseases, as we seek to develop a new class of therapies for patients with high unmet need.” “For nearly 30 years, Théa has been committed to bringing the most modern and diverse range of innovative ophthalmic products to the market for the benefits of eye care practitioners and patients. We are very excited to continue the development of sepofarsen and ultevursen for patients,” said Jean-Frédéric Chibret, President of the Théa group. “These two programs can deliver hope for patients suffering from retinal diseases that lead to blindness. We look forward to returning these assets into the clinic.” Within Théa, a fully dedicated team specializing in inherited retinal disorders and a new organization are currently being set up to manage these two projects. More information on the next steps for these programs will be available in the coming weeks from Théa. ProQR’s financial advisor is Lazard, with Allen & Overy acting as legal advisor. Théa’s legal advisor is Dentons. The transaction is expected to close in the third quarter of 2023, subject to the satisfaction of certain closing conditions. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer®, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. About Théa Théa is the leading independent European pharmaceutical company specialized in the research, development, and commercialization of eye care products. Based in Clermont-Ferrand, France, this family-owned and run company comprises more than 1700 collaborators and has expanded by opening more than 35 affiliates and offices in Europe, North Africa, North and South America, and the Middle East. Its products are available in 75 countries. In 2022, Théa’s turnover reached $941 million. Learn more about Théa at https://www.laboratoires-thea.com. About Sepofarsen Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. The mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen 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 and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA. About Ultevursen Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. Ultevursen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA. About Axiomer® ProQR is pioneering a next-generation RNA base editing technology called Axiomer®, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer® EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. 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. Such forward-looking statements include, but are not limited to, statements regarding this divestment, the potential payments and earnouts arising out of the divestment, the expected timing for the closing of the divestment, the further development of sepofarsen and ultevursen, as well as the potential of our technologies and product candidates. 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. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, 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. These risks and uncertainties include, among others, the clinical development activities to be performed by Théa and the condition of successful market access for sepofarsen and ultevursen; the cost, timing and results of preclinical studies and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; our reliance on contract manufacturers or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Eli Lilly and Company; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; and general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties. 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. For ProQR Therapeutics N.V. Investor contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 skiely@proqr.com or Hans Vitzthum LifeSci Advisors T: +1 617 430 7578 hans@lifesciadvisors.com Media contact: Robert Stanislaro FTI Consulting T: +1 212 850 5657 robert.stanislaro@fticonsulting.com For Théa Julie Ahmed Communications Manager Julie.ahmed@theapharma.com

Uni-Rare Study will improve clinical understanding of more IRDs and boost development of potential therapies. COLUMBIA, Md., Nov. 10, 2022 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, is committing at least $8.6 million for its Uni-Rare Study, a new natural history study for approximately 1,500 people with one of more than 300 rare genes associated with inherited retinal diseases (IRDs) including: retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions. The study, due to begin recruitment in December, will help clinical researchers gain a better understanding of the course of retinal degeneration and vision loss for people with mutated genes that have not been well-characterized in the clinic. The study will also help the IRD research and therapeutic development community: 1) identify more people for clinical trials of therapies, 2) inform design of clinical trials, and 3) identify therapeutic targets for more people. "A majority of the 300 genes linked to inherited retinal diseases have not been well characterized in the clinic. That is, we do not have a good understanding of retinal degeneration and disease progression for those affected by these mutated genes," says Todd Durham, PhD, senior vice president, clinical development, at the Foundation Fighting Blindness. "We are excited to launch a highly inclusive study to benefit a large segment of the population affected by IRDs, which are so genetically diverse." "We are at a promising juncture in the clinical development of genetic therapies for inherited retinal diseases," says José-Alain Sahel, MD , distinguished professor and chairman of the department of ophthalmology at the University of Pittsburgh School of Medicine and Uni-Rare Study chair. "With dozens of emerging treatments in, or moving toward, clinical trials, the Uni-Rare Study will be invaluable in defining and validating outcome measures for clinical trials and establishing the infrastructure for multicenter studies. The Foundation Fighting Blindness, with its strong presence and relationships in the global IRD research community, is the ideal partner to lead the effort." The two-part Uni-Rare Study will be conducted by the Foundation's Clinical Consortium, a global, 40-site network of clinical research centers equipped with the experts and resources needed to launch IRD clinical trials and natural history studies. The Jaeb Center for Health Research will serve as the coordinating center. In the first part of the study, all participants will have an initial evaluation to collect their genetic data and clinical measures. When the study is initiated, study participants whose IRD is caused by variants in the RDH12 and MYO7A genes may qualify for the second part of the study, which involves collecting clinical measures for an additional four years. Additional gene cohorts for this part of the study are expected to be added in the future. The Foundation has received support for the study from families affected by Usher syndrome type 1B, Save Sight Now, Opus Genetics, Atsena Therapeutics, and Cove Therapeutics and is seeking additional support to add further gene cohorts to the second part of the study. "Opus is pleased to be an early supporter of this first-of-its-kind in ophthalmology initiative to understand natural histories around rare diseases of the retina in order to accelerate research and therapeutic development for these blinding conditions," said Jennifer Hunt, chief development officer at Opus Genetics. "Atsena Therapeutics is pleased to support the Uni-Rare natural history study and the inherited retinal disease community as we continue advancing the preclinical development of our dual AAV vector-based gene therapy to prevent blindness from USH1B," said Kenji Fujita, MD, chief medical officer at Atsena Therapeutics. "We are proud to sponsor such a robust interrogation of natural history that will be used to inform future clinical development plans and improve patient outcomes; these data will be fundamental for our understanding of several diseases and will serve to accelerate the development of Cove's non-viral gene therapies," said Niren Shah, PharmD, MBA, CEO and co-founder of Cove Therapeutics. IRD patients and physicians with IRD patients can get more information at clinicaltrials.gov. Patients with IRDs caused by mutations in the following genes are not eligible for the study: ABCA4, CEP290, CHM, CNGA3, CNGB3, EYS, GUCY2D, PCDH15, PROM1, RHO, RPE65, RPGR, USH2A. These genes are or have been part of other natural history studies or clinical trials. About the Foundation Fighting Blindness Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. About Opus Genetics Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness's venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit opusgenetics.com. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company's ongoing Phase I/II clinical trial is evaluating a potential therapy for a form of LCA, one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina's Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. About Cove Therapeutics Cove Therapeutics is a privately held biotechnology company focused on developing non-viral, non-lipid, nanoparticle gene therapies for orphan and chronic prevalent indications. The Company utilizes a nanoparticle technology that overcomes limitations in gene therapy by enabling cell-specific tropism, scalable manufacturing, redosing, and packaging of large genes while maintaining a favorable safety profile. Together with these nanoparticles, Cove's lead franchise for ophthalmic conditions leverages a novel outpatient suprachoroidal device that enables broad distribution of therapeutics. Learn more about the Company through direct contact — Stephen Jasper, Gilmartin Group, [email protected], 858-525-2047. Media Contact: Chris Adams Vice President, Marketing & Communications [email protected] 410-423-0585 SOURCE Foundation Fighting Blindness