The U.S. FDA is to temper the alert it put out in November 2023 pointing to a potential risk of CAR T therapies causing de novo malignancies. “There was this issue of possible safety concerns with T-cell lymphomas, with these CAR T cells. I think this year, we are feeling reassured in this regard,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), told the Alliance for Regenerative Medicine briefing at the J. P. Morgan Healthcare Conference on Jan. 13.
The accelerating pace of U.S. FDA approvals for cell and gene therapies is “great for the field and great news for the patients,” but questions remain over commercialization, with “costs remaining stubbornly high.” That was the glass half-full summary of Tim Hunt, president of the industry group, the Alliance for Regenerative Medicine, reprising progress in 2024, and looking forward to the prospects for further growth and the potential impact of the incoming Trump administration in 2025.
U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
The U.S. FDA is promising to make 2024 a “breakout” 12 months for gene therapies, with a number of initiatives to promote clinical development, approvals and uptake. “This is a great year to focus on gene therapy,” said Peter Marks, director of the Center for Biologics Evaluation and Research at the FDA. “I just want to focus on moving ahead gene therapy,” he told attendees of the J. P. Morgan Healthcare Conference on Jan. 8.
With the massive amounts of capital raised by global public and private biopharmaceutical companies last year generating approximately $134 billion – a total that was almost double the previous record of about $69 billion raised in 2015 – it is not surprising that financing for the regenerative medicine and advanced therapy sector also set an annual record.
A third-quarter progress report from the international advocacy group Alliance for Regenerative Medicine (ARM) has determined that the regenerative medicine and advanced therapy (RMAT) sector established a highwater mark of $15.9 billion in global financings, breaking the previous annual record of $13.5 billion that was set in 2018.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine (ARM), finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising despite the challenges posed by the ongoing COVID-19 pandemic.
The Rare Disease Day, which takes place at the end of February each year, is designed to focus global attention on the need for therapies to treat patients suffering from devastating rare diseases. The most recent event represented the 13th year it has been held. Over that period, research and development in the area has come a long way, and there are now 420 companies around the world that are active in developing regenerative medicines and advanced therapies for the treatment of rare diseases, according to a new report released by the Alliance for Regenerative Medicine (ARM).
