Business:
Gene Therapy
Drug notes:
4D-150 Clin1/Clin2 wet AMD, Clin0 DME; 4D-125 Clin1/Clin2 XLRP; 4D-110 Clin1/Clin2 choroideremia; 4D-710 Clin1/Clin2 cystic fibrosis, additional effort Clin0 cystic fibrosis; 4D-175 RD geographic atrophy; 4D-725 RD A1AT deficiency; undisclosed RD eye disease
About:
4D Molecular Therapeutics is designing and developing transformative gene therapies for unmet medical conditions. Adeno-associated viruses (AAVs) have great potential for targeted delivery and efficient transduction of gene therapies. 4D is using their Therapeutic Vector Evolution platform to discover custom and proprietary AAVs for specific tissues and diseases to build a diverse vector library. Selected vectors are chosen and tested in non-human primates based on their Target Vector Profile for a disease. Subsequent testing is performed in human cell and organotypic tissue assays. 4D is developing a diverse pipeline of product candidates for cardiology, pulmonology and ophthalmology with products in early clinical trials.
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