![]() 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. The two companies will jointly identify clinical development candidates, after which Astellas will have an exclusive option to licence the rights for further development and commercialisation of any discovered compounds.At 4D Molecular Therapeutics, Inc. The partnership, which is worth up to $1.9bn, will utilise Cullgen’s uSMITE targeted protein degradation platform, featuring novel E3 ligands, alongside Astellas' drug discovery capabilities. The deal comes just less than a month after Astellas and Cullgen entered into a research collaboration and exclusive option agreement aimed at advancing targeted protein degraders. “This collaboration with Astellas, a leader in AAV gene therapy, continues to validate R100 for routine intravitreal low dose delivery of genetic payloads for the treatment of retinal diseases,” he said.Īdam Pearson, chief strategy officer at Astellas, said: "We believe that this collaboration will bring synergies between the two companies' cutting-edge research, and will ultimately lead to the development of new therapeutics for patients with ophthalmic diseases at high risk of blindness.” The Japanese drugmaker will be responsible for all research, development, manufacturing, and commercialisation activities for the resulting programmes.ĭavid Kirn, co-founder and chief executive officer of 4DMT, outlined that over 70 patients have been dosed with R100-based product candidates in wet age-related macular degeneration and rare ophthalmic diseases. According to the companies, it "has the ability to penetrate the internal limiting membrane barrier and to efficiently transduce the entire retina, resulting in robust transgene expression within retinal cells".Īstellas will use the vector initially for one genetic target implicated in rare monogenic ophthalmic diseases and will have the option to add up to two more targets after paying additional option exercise fees. R100 is an adeno-associated virus vector invented by 4DMT for intravitreal delivery. ![]() Under the terms of the licensing agreement, the US genetic medicines company will receive an upfront payment of $20m in exchange for its proprietary R100 vector technology and will also be eligible for future option fees and milestones of up to $942.5m plus certain royalties. Astellas Pharma has said it will be gaining the rights to use one of 4D Molecular Therapeutics’ (4DMT) gene therapy vectors for at least one rare eye disease.
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