The CF Foundation’s investment is being offered as part of its $500 million Path to a Cure initiative to accelerate development of treatments that address the underlying cause of the disease and a cure for CF. The CF Foundation previously awarded 4DMT over $3 million in funding to support discovery and development of AAV gene delivery to the lung in 2016. 4DMT retains worldwide rights to develop and commercialize 4D-710 and any other potential medicines. 4DMT will match the funds provided and allocate these funds to the ongoing development of 4D-710. Under this agreement, the CF Foundation will support the completion of IND-enabling research and development activities, and the planned Phase 1/2 clinical study of 4D-710, 4DMT’s wholly-owned product candidate for the aerosol treatment of CF lung disease.Īs part of this agreement, the CF Foundation has committed $14 million in new funding, consisting of a $10 million equity investment in 4DMT’s recently completed Series C financing and additional funding contingent upon achievement of a pre-clinical development milestone. The CF Foundation’s investment builds upon previous work with 4DMT to advance new therapies for CF. – J– 4D Molecular Therapeutics (4DMT), a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, today announced a new agreement with Cystic Fibrosis Foundation (CF Foundation) to develop precision gene medicines for cystic fibrosis (CF). This internal capability and close collaboration between our R&D and Manufacturing teams has greatly accelerated the pace of discovery at 4DMT.EMERYVILLE, CA. In addition to TVE, our technology includes a robust AAV manufacturing platform and onsite manufacturing facility that allows us to rapidly produce and test research grade material and scale up to GMP clinical material. We believe we are positioned to create, develop, manufacture and, if approved, effectively commercialize targeted genetic medicines that could transform the lives of patients suffering from debilitating diseases. We have built a robust and efficient product engine with 6 open Investigational New Drug Applications (INDs) in the U.S., 1 IND in Taiwan, and 1 Clinical Trial Approval (CTA) in Australia. We believe this validates the power of our directed evolution platform for discovering superior vectors compared to wildtype viral vectors. To-date, we have demonstrated clinical proof-of-concept for three evolved vectors in three therapeutic areas and routes of administration with five products and patient populations. In addition, we have two product candidates in preclinical studies: 4D-175 for geographic atrophy (GA) and 4D-725 for alpha-1 antitrypsin deficiency. We have built a deep portfolio of AVV-based gene therapy product candidates, with five product candidates in clinical trials: 4D-150 for the treatment of wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME), 4D-710 for the treatment of cystic fibrosis lung disease, 4D-310 for the treatment of Fabry disease cardiomyopathy, 4D-125 for the treatment of X-linked retinitis pigmentosa (XLRP), and 4D-110 for the treatment of choroideremia. These profiles will allow us to treat a broad range of large market diseases, unlike most current genetic medicines that generally focus on rare or small market diseases. We believe key features of our targeted and evolved vectors will help us create targeted product candidates with improved therapeutic profiles. We seek to unlock the full potential of gene therapy using our platform, Therapeutic Vector Evolution (TVE), which combines the power of directed evolution with our approximately one billion synthetic AAV capsid-derived sequences to invent evolved vectors for use in our products. We are committed to breaking boundaries and daring to cure as we develop new and foundational products and product components through our growing technology platforms.ĤDMT is a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines. (“4DMT”), we boldly innovate to unlock the full potential of genetic medicines for countless patients.
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