Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.
2021 could set a record in new product approvals in regenerative medicines and advanced therapies; alongside unprecedented levels of financing. And it’s a trend that looks to continue given the volume of trials ongoing worldwide, according to the Alliance for Regenerative Medicine (ARM).
Gene therapy is having a moment. Once confined to academic labs and a couple of companies, the field now encompasses hundreds of trials, dozens of companies and billions of investment dollars. With greater power, though, comes greater responsibility. The FDA is asking a panel of outside experts to try and define those responsibilities as they debate a lengthy list of 15 questions Thursday and Friday, in a two-day marathon meeting on the future of a field that has promised one-time treatments for some of the most famous and fatal diseases known to medicine.
The first half of 2021 was a very productive period for companies developing cell and gene therapies and other regenerative medicines. A total of 2,648 clinical trials were underway at the end of this period, almost evenly divided between industry and academic and government sponsors. Of this number, 243 trials were in Phase 3 – a sign of the maturing of the pipeline, according to the Alliance for Regenerative Medicine (ARM). ARM published the clinical data, along with regulatory and financial information about the sector, on 18 August.
A planned review of the EU’s legislation covering blood, tissue and cells (known as BTC) is a source of tension for developers of Advanced Therapy Medicinal Product (ATMPs), said Paige Bischoff Paige Bischoff, senior vice president of global public affairs at the Alliance for Regenerative Medicine. The rules regulate the use of human-derived substances like donated bone marrow or sperm and eggs used in in vitro fertilization.
Industry experts warn that Europe risks sitting out on the transformation as it loses ground to competitors in the U.S. and China. A fractured market and a host of regulatory obstacles mean that setting up in the bloc isn’t an easy task, especially when negotiating 27 different sets of rules.
The global regenerative medicine industry is growing rapidly, and the Asia-Pacific region is a major center of growth for the sector driven primarily by the recently added new Korean and Taiwanese clinical trials, a report said.
Biotech developers of cell, gene and regenerative therapies raised $14 billion in funding over the first six months of 2021, an enormous sum that equals roughly three-quarters of last year’s record total of $19.9 billion. Venture capital firms have been particularly active, according to numbers compiled by the industry association Alliance for Regenerative Medicine in a new report, pouring more than $5 billion into funding rounds for private biotech companies in the field.
