4D Molecular Therapeutics Raises $75 Million to Advance Gene Therapy Programs

4D Molecular Therapeutics raised $75 million in a series C financing to advance a pipeline of AAV gene therapies targeting rare diseases.

Viking Global Investors led the financing, with participation from new investors including Amzak Health, Casdin Capital, Cystic Fibrosis Foundation, Longevity Vision Fund, MiraeAsset Financial Group, Octagon Investments, and QUAD Investment Management. Existing investors also participated in the financing, including Arrowmark Partners, Berkeley Catalyst Fund, BVF Partners, Pappas Capital and Chiesi Ventures, Perceptive Advisors, Pfizer Ventures, and Ridgeback Capital Investments.

4D Molecular (4DMT) is developing precision-guided AAV gene medicines based on directed evolution. The biotech’s proprietary Therapeutic Vector Evolution platform enables customization of AAV vectors to target specific tissue types associated with the underlying disease. These optimized AAV vectors are designed to provide targeted delivery by routine clinical routes, efficient transduction, reduced immunogenicity, and resistance to pre-existing antibodies—attributes that the company says could enable the development of gene therapies that overcome known limitations of conventional AAV vectors and enable 4DMT to pursue previously untreatable patient populations and address a broad range of rare and large market disease markets.

Proceeds from the financing will be used to advance three of 4DMolecular’s precision-guided AAV gene therapy product candidates through initial clinical proof-of-concept in patients, to advance it’s proprietary pipeline and next-generation Therapeutic Vector Evolution platform, and to expand its internal GMP manufacturing capabilities.

Before the end of the year, 4DMT expects to initiate clinical trials for 4D-310, its wholly owned product candidate for the treatment of Fabry disease, and two ophthalmology product candidates: 4D-125 for the treatment of X-linked retinitis pigmentosa, a wholly owned candidate subject to an exclusive option for Roche to develop and commercialize, and 4D-110, a candidate for the treatment of choroideremia that is licensed to Roche.

The financing will also help support the biotech’s preclinical pipeline and platform, including IND-enabling studies for 4D-710, it’s experimental aerosol treatment of cystic fibrosis lung disease, and candidates in neuromuscular diseases and ophthalmology for both rare and large markets.

“This financing enables 4DMT to advance three product candidates into clinical trials, and to progress our mission of unlocking the full potential of gene therapy for broad populations of patients suffering from both rare and large market diseases,” said David Kirn, co-founder, chairman and CEO of 4D Molecular Therapeutics.

Photo: David Kirn, co-founder, chairman and CEO of 4D Molecular Therapeutics

Author: Rare Daily Staff