Patisiran sodium

CAMBRIDGE, United Kingdom and MONTREAL and VANCOUVER, British Columbia and BASEL, Switzerland, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Epitopea, a transatlantic cancer immunotherapeutics company developing accessible off-the-shelf RNA-based immunotherapies, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and a robust and expansive lipid nanoparticle (LNP) patent portfolio, today announced that they have entered into a collaboration and nonexclusive license agreement to develop novel mRNA-LNP immunotherapies targeting Epitopea’s proprietary aberrantly-expressed tumor specific antigens (aeTSAs), called CryptigensTM, for an undisclosed oncology indication. “At Epitopea we continue to accelerate our near-term clinical development plans for our next generation, tumor selective, off-the-shelf, RNA-based immunotherapies that we believe have the potential to extend the durability of clinical responses in patients. Access to world-class LNP delivery technology in support of our vision is critical. Genevant is a longstanding leader in the LNP space, and we are excited about the translational path forward,” said Alan Rigby, Chief Executive Officer of Epitopea. “Our clinically validated LNP technology and decades of experience in the space make us a partner of choice for innovative RNA-based immunotherapy companies,” said James Heyes, Chief Scientific Officer of Genevant Sciences. “We are delighted to be collaborating with the Epitopea team and supporting its mission to extend the durability of clinical response in cancer patients.” Under the terms of the agreement, Genevant granted to Epitopea a nonexclusive worldwide license to certain Genevant LNP technology to develop RNA-based immunotherapies targeting Epitopea’s Cryptigen™ TSAs in an undisclosed oncology indication. Genevant is eligible to receive up to $123.5 million in upfront and contingent milestone payments per product and tiered royalties ranging from the mid to high single digits on future product sales. Contact information EpitopeaDr. Alan C. Rigby – CEOAlan.Rigby@epitopea.com Scius Communications Katja Stout+44 7789 435990katja@sciuscommunications.com Daniel Gooch+44 7747 875479Daniel@sciuscommunications.com Genevant Sciencespete.zorn@genevant.com About Epitopea Epitopea is a transatlantic cancer immunotherapeutics company developing accessible off-the-shelf RNA-based immunotherapies for use in hard-to-treat cancers by targeting a new class of untapped tumor-specific antigens, which are known as Cryptigen™ TSAs, that are broadly shared across multiple patients with the same tumor type. Epitopea has created an extensive library of novel Cryptigen™ TSAs, discovered by its proprietary CryptoMap™ platform that leverages immunopeptidomics, genomics, and a bioinformatics pipeline, allowing the identification of aberrantly expressed, tumor-specific antigens (aeTSAs) that are hidden within cancer’s ’junk’ DNA. These hidden Cryptigen™ TSAs were first discovered through research led by Drs. Claude Perreault and Pierre Thibault at the Institute for Research in Immunology and Cancer at the Université de Montréal. Epitopea is backed by leading life science investors including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Le Fonds de Solidarité FTQ, Investissement Québec, adMare BioInnovations, Jonathan Milner, the Harrington Discovery Institute, IRICoR and Novateur Ventures. Epitopea was founded in 2021 and consists of sister companies based in Cambridge, UK and in Montreal, Canada. For additional information, please visit www.epitopea.com and follow Epitopea on LinkedIn. About Genevant Sciences Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, a robust and expansive lipid nanoparticle (LNP) patent portfolio, and decades of experience and expertise in nucleic acid drug delivery and development. Genevant’s scientists have pioneered LNP delivery of nucleic acids for over 20 years, and Genevant’s LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved systemic RNA-LNP product (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. For more information, please visit www.genevant.com.

Industry veterans John Maraganore, Ph.D., and Rachel Meyers, Ph.D., to provide strategic guidance on the company’s RNA Actuating platform to develop a new class of RNA medicines that upregulate protein coding genesCAMBRIDGE, Mass., Dec. 10, 2024 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (Nasdaq: CAMP), a clinical-stage biotechnology company developing a pipeline of regRNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels across a range of genetic diseases, today announced the appointments of John Maraganore, Ph.D., and Rachel Meyers, Ph.D., as strategic advisors to the Company. For nearly 20 years, Dr. Maraganore served as the founding Chief Executive Officer and Director of Alnylam where he led the company’s programs in RNA interference through global commercialization, resulting in the launch of the first four RNAi therapeutics, ONPATTRO®, GIVLAARI®, OXLUMO®, and LEQVIO®. Additionally, Dr. Meyers brings more than two decades of life sciences leadership, research, and development experience in RNA-based medicines, having spent nearly 14 years at Alnylam where she most recently served as Senior Vice President of Research and RNAi Lead Development. She currently sits on the company’s Scientific Advisory Board. “We feel privileged and are delighted to welcome Drs. Maraganore and Meyers to our CAMP4 team as strategic advisors,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “We view their joining the team as a testament to the promise and potential of our approach for upregulating gene expression to potentially address a broad range of diseases. As we prepare for 2025, we look forward to benefiting from their insights and expertise as we advance our efforts in bringing potentially transformative RNA medicines to patients in need of disease-modifying solutions.” Dr. Maraganore added, “The targeted upregulation of gene expression has been a long-standing challenge for the field, and by applying established and clinically validated antisense oligonucleotide technology to groundbreaking science, CAMP4 could potentially make a meaningful impact on patients across a broad range of diseases. I look forward to working with them as they pioneer the field of upregulation.” Prior to his role at Alnylam, Dr. Maraganore led the product franchises in oncology, cardiovascular, inflammatory, and metabolic diseases for Millennium Pharmaceuticals, in addition to leadership of M&A, strategy, and biotherapeutics. Earlier in his career, Dr. Maraganore invented and spearheaded the development of ANGIOMAX® (bivalirudin) for injection while at Biogen and was also a scientist at ZymoGenetics, Inc., and the Upjohn Company. He currently serves as a Venture Partner at ARCH Venture Partners, a Venture Advisor at Atlas Ventures, an Executive Partner at RTW Investments, a Senior Advisor for Blackstone Life Sciences, and a Senior Advisor for Jeffries Financial Group. Beyond his leadership roles in the investment community, Dr. Maraganore also sits on the Board of Directors for multiple publicly traded companies, including Beam Therapeutics, Kymera Therapeutics, Rapport Therapeutics, and Takeda Pharmaceuticals. He received his B.A., M.S., and Ph.D. in biochemistry and molecular biology at the University of Chicago. Dr. Meyers currently serves as a member of the Board of Directors of Korro Bio (Nasdaq: KRRO), a biopharmaceutical company focused on discovering, developing, and commercializing a new class of genetic medicines based on editing RNA, and also holds a variety of advisory roles within the biotech community. Most recently, she served as Founder and Chief Scientific Officer at Faze Medicines, a Third Rock Ventures-spawned biotech company focused on treating diseases of high unmet need through the perturbation of biomolecular condensates. Earlier in her career, Dr. Meyers was a senior scientist at Millennium Pharmaceuticals and today serves on multiple scientific advisory boards, including the National Advisory Board on Innovation and Entrepreneurship through the Department of Commerce. Furthermore, she is listed as an inventor on numerous patents and patent applications and has authored many peer-reviewed publications. Dr. Meyers received her postdoctoral training with Lew Cantley at Harvard Medical School in signal transduction and earned her Ph.D. in biology from Nobel laureate Phil Sharp at the Massachusetts Institute of Technology in in vitro transcription. She earned her BA in Biochemistry from Brandeis University. “I’ve had the unique opportunity to work alongside tremendously talented professionals who have led the shaping of the RNA medicines space,” said Dr. Meyers. “Having been at the forefront of these scientific breakthroughs, I believe now more than ever RNA-based therapies are poised to continue transforming how we treat patients with complex diseases. Having the opportunity to advise CAMP4 as they continue advancing the field is an extension of the work to which I’ve committed my career.” About CAMP4 Therapeutics CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic ASO drug candidates target regRNAs, which act locally on transcription factors and are the master regulators of gene expression. CAMP4’s proprietary RAP Platform™ enables the mapping of regRNAs and generation of therapeutic candidates designed to target the regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. Learn more about us at www.CAMP4tx.com and follow us on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning CAMP4’s plans, objectives, expectations and intentions; the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting SAD and MAD data from and seeking regulatory approval for the CMP-CPS-001 trial; its growth strategy; and cash balance guidance. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other information we file with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contacts Investor Relations:Sandya von der WeidLifeSci Advisorssvonderweid@lifesciadvisors.com Media:Jason Braco, Ph.D.LifeSci Communicationsjbraco@lifescicomms.com