4D Molecular Therapeutics is a clinical-stage precision gene medicines company that harnesses directed evolution to develop precision-guided AAV gene therapies. Its proprietary Therapeutic Vector Evolution platform enables a disease-first approach to customize AAV vectors to target specific tissues, aiming for targeted delivery, efficient transduction, reduced immunogenicity, and resistance to pre-existing antibodies. The company focuses on lysosomal storage diseases, ophthalmology, neuromuscular diseases, and cystic fibrosis and pursues both rare and large-market indications. Proceeds from the recently closed financing will be used to advance multiple product candidates through initial clinical proof-of-concept, expand internal GMP manufacturing capabilities, and progress the pipeline and next-generation platform. 4DMT expects to initiate clinical trials in 2020 for 4D-310 (Fabry disease), 4D-125 (X-linked retinitis pigmentosa, subject to an exclusive option for Roche), and 4D-110 (choroideremia, licensed to Roche), and is supporting IND-enabling studies for 4D-710 (aerosol treatment for cystic fibrosis). The company designs vectors to enable routine clinical routes and improved therapeutic profiles to address previously untreatable patient populations. 4D Molecular Therapeutics develops targeted, customized next-generation adeno-associated virus (AAV) gene therapy products using its Therapeutic Vector Evolution discovery platform. The platform applies principles of evolution and natural selection across over 100 million unique AAV variants from more than 35 proprietary libraries to identify vectors that target specific tissues via clinically optimal routes at lower doses. 4DMT engineers vectors with resistance to pre-existing antibodies to improve delivery and efficacy across organs and disease indications. The company intends to treat both rare genetic diseases and larger-market conditions through internal programs and partnered programs. Its lead intravitreally delivered AAV gene therapy candidate for choroideremia is expected to enter clinical trials in 2019. Proceeds from the recent financing will be used to advance the Therapeutic Vector Evolution platform and the company’s pipeline. 4DMT uses a Therapeutic Vector Evolution discovery platform to create proprietary AAV vectors optimized for specific target cells, organs, routes of administration, and antibody evasion. Its lead pulmonary program, 4D-710, employs an AAV vector designed to deliver CFTR to lung airway cells via aerosolized delivery. The company is advancing multiple internal products toward clinical trials while also partnering with biopharma companies including Pfizer, Roche, uniQure, AGTC and Benitec. 4DMT says its platform deploys roughly 100 million unique AAV variants and applies selection methods to identify highly optimized vectors. Management includes co-founders David Kirn (CEO) and David Schaffer (acting CSO), who emphasize collaboration with clinicians and patient advocates. The article reports targeted IND-enabling work for 4D-710 supported by non-dilutive funding from the Cystic Fibrosis Foundation Therapeutics.

Total raised

$168M

Funding rounds

3

Latest round

Series C

Latest activity

Jun 2020