Investment in cell and gene therapies skyrocketed during the first half of 2021 to $14.1bn, the highest six-month total on record and up 35% from the first half of 2020, which was a record-breaking year, according to a new report from the Alliance For Regenerative Medicine (ARM).
For a long time, gene and cell therapies were a treatment option for the future. Now, the pace of development is moving at breakneck speed, with a number of firsts and $14.4 billion in financing collected over the initial half of the year. The Alliance for Regenerative Medicine has documented the “acceleration on all fronts” in a new report looking at the first half of 2021
The European Commission’s latest consultation on overhauling the EU legislation on medicines for children and for rare diseases has drawn some sharp responses from stakeholders. The Alliance for Regenerative Medicine (ARM), which represents companies developing advanced therapy medicinal products (ATMPs), believes that a legal definition of unmet medical needs was not necessary.
The Alliance for Regenerative Medicine, an industry group representing developers of gene and cell therapies, also gave its input to the commission consultation. These advanced therapies are often targeted at rare diseases. Unsurprisingly, ARM was similarly resistant to change in the regulation. “Changes in the regulation without adding specific incentives would not increase R&D and investments in rare diseases,” writes ARM. The organization adds that clinical trials currently present a challenge for developing products for small target populations, given the difficulty in gathering enough patients for a proper randomized clinical trial.
The Alliance for Regenerative Medicine welcomed the advisory committee meeting and the opportunity for comment through the public docket, which is open until 26 August. “We view it as a positive development for the FDA’s regulatory approach that the agency is seeking input from the advisory committee and soliciting comments from the public,” said Stephen Majors, ARM’s director of public affairs.
Lawmakers are hewing closely to President Joe Biden’s proposal for a new $6.5 billion science agency as part of a sweeping measure to deliver medical breakthroughs.
Reps. Diana DeGette (D-Colo.) and Fred Upton (R-Mich.) on Tuesday released a draft bill, dubbed Cures 2.0 — the follow-up to the landmark 21st Century Cures Act biomedical innovation law (Public Law 114-255). The legislation’s release coincides with a commentary in Science magazine setting forth the vision of White House science office and National Institutes of Health officials for the Advanced Research Projects Agency for Health, as well as why it would be different from existing programs and centers.
A bill making its way through the German legislative system could make it easier for hospitals to secure financing for cell and gene therapies. The Alliance for Regenerative Medicine (ARM) has welcomed the move and says it will “reduce a bureaucratic hurdle that stood in the way of patient access to durable and potentially curative” advanced therapy medicinal products.
