![]() ![]() Please refer to your health care provider(s) for your individualized, specific care management needs. The intent of this shared content is to serve as a point of reference for informational purposes. CureDuchenne does not provide medical advice nor is CureDuchenne a medical provider. Please be advised that the contents within the CureDuchenne site include a compilation of articles, advisements, and references from the community. PRIVACY POLICY | TERMS OF USE | INDIRECT COST POLICY | TAX INFOĬureDuchenne is a 501c3 registered tax-exempt nonprofit organization. Therapeutic Vector Evolution empowers 4DMT to create customized gene therapy products to deliver genes specifically to any tissue or organ in the body, by the optimal clinical route of administration for any given disease, at lower doses and with resistance to pre-existing antibodies. The Duchenne community is aware of possible limitations of the current delivery system for gene therapy and gene editing due to pre-existing neutralizing antibodies, and manufacturing challenges due to the large amount of vector needed to treat the entire muscular system in the body.ĤDMT focuses on the discovery and development of targeted, customized and proprietary next-generation AAV gene therapy products for use in patients with severe genetic diseases with high unmet medical need. “CureDuchenne’s investments are moving quickly through the drug development process and we look forward to safety and efficacy data for these potential therapies.” So far, we have had preclinical success in this area for Duchenne with our investments in duplication 2 exon skipping at Nationwide Children’s Hospital, Bamboo Therapeutics/Pfizer and Exonics Therapeutics, and we are very positive about the prospect of 4DMT’s work,” said Debra Miller, founder and CEO of CureDuchenne. “Our investment into 4DMT will be the fourth investment by CureDuchenne Ventures, the investment arm of CureDuchenne, into gene therapy and gene editing. ![]() – ( BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, in an effort to advance gene therapy. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.NEWPORT BEACH, Calif. No representation is made as to the safety or effectiveness of 4D-150, 4D-310, 4D-710, 4D-125, and 4D-110 for the therapeutic use for which they are being studied. The five 4DMT product candidates in clinical development are: 4D-150 for wet AMD, 4D-310 for Fabry disease, 4D-710 for cystic fibrosis, 4D-125 for XLRP, and 4D-110 for choroideremia.ĤD-150, 4D-310, 4D-710, 4D-125, and 4D-110 are in clinical trials and have not yet been approved for marketing by the US FDA or any other regulatory authority. The 4DMT targeted and evolved vectors are invented with the goal of being delivered at relatively low doses through clinically routine, well tolerated and minimally invasive routes of administration, transducing diseased cells in target tissues efficiently, having reduced immunogenicity and, where relevant, having resistance to pre-existing antibodies. The company is initially focused on five clinical-stage products in three therapeutic areas for both rare and large market diseases: ophthalmology, cardiology (including Fabry disease) and pulmonology. 4DMT seeks to unlock the full potential of genetic medicines using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent targeted and evolved vectors for use in our products. ETĮvent: Chardan’s 6 th Annual Genetic Medicines ConferenceĪ live audio webcast of the presentation will be available by visiting the “Investors & Media” section of the 4DMT website at A replay of the webcast will be available for at least two weeks following the live event.ĤDMT is a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines. (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced that management will participate in the following upcoming investor conferences:Įvent: Jefferies Cell and Genetic Medicine Summitĭate & Time: Friday, September 30, 2022, at 9:00 a.m. 20, 2022 (GLOBE NEWSWIRE) - 4D Molecular Therapeutics, Inc. ![]()
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