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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.
“Our investment into 4DMT will be the fourth investment by CureDuchenne Ventures, the investment arm of CureDuchenne, into gene therapy and gene editing. 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. “CureDuchenne’s investments are moving quickly through the drug development process and we look forward to safety and efficacy data for these potential therapies.”
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.
4DMT 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. 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.
CureDuchenne is the leading nonprofit dedicated to extending and improving the lives of 300,000 boys worldwide who have Duchenne muscular dystrophy, a degenerative disease with no known cure. CureDuchenne uses an entrepreneurial venture philanthropy model to fund impactful research and accelerate human clinical trials. Through its CureDuchenne Cares program, CureDuchenne provides confidence to families seeking valuable resources, trains physical therapists and other health professionals on new standards of care and provides hope, support and empowerment to the Duchenne community. For more information, please visit www.cureduchenne.org, and follow us on Facebook, Twitter and YouTube.