GlobalData, a data and analytics company, on Tuesday said that Sangamo is expected to gain a significant market share, as its therapy will likely achieve approval before 4DMT’s therapy.
4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and cardiovascular, pulmonary, and ophthalmic diseases, has recently been issued a clinical hold by the FDA for its Fabry Disease (FD) gene therapy program (4D-310). 4DMT and Sangam Therapeutics are the leading developers in the Fabry disease space, and with the FDA’s clinical hold, the future of 4DMT’s program remains uncertain.
4DMT paused the enrolment of patients to two of its trials for 4D-310 in January 2023, following significant adverse events where three patients experienced kidney issues; however, these were treated and resolved in a four-week period.
“The 4DMT trial, however, presents challenges and barriers to the development of gene therapy as significant adverse events are still occurring in late-stage therapies, and this may present a deterrent for the development of further gene therapies in the long-term. This setback for 4DMT, although downplayed by the company in the short-term, will likely lead to Sangamo’s therapy becoming the lead FD gene therapy in the pipeline,” Akash Patel, Pharma Analyst at GlobalData, said.
4DMT is creating a gene therapy using an adeno-associated viral vector (AAV) and there is currently competition from several early-stage therapies in trial, such as Sangamo’ AAV-based Fabry gene therapy that is progressing to Phase III.
“FD will likely become a disease that is treatable with gene therapy; however, barriers to access remain with late-stage trials to be completed, followed by likely issues surrounding pricing, insurance coverage, and reimbursement across the major markets in the US and Europe,” Patel added.
