The Alliance for Regenerative Medicine reported on 22 November that cell and gene therapy companies have raised enough money already in 2021 to break the record set in 2020. Venture capital financings are driving the surge with $8.2bn raised through the third quarter, which is up 45% from the same nine-month period last year.
Improving drug competition and boosting patient access to innovative treatments will likely be among the priorities of Robert Califf at the helm of the Food and Drug Administration, policy analysts say.
Cell and gene therapy sponsors could see some submissions gain attention at the US Food and Drug Administration sooner that those already on file if they are deemed more important to the development program. Center for Biologics Evaluation and Research Director Peter Marks said staff are working on a triage process for some submissions, apparently so product development does not slow unnecessarily.
In response to the US Food and Drug Administration’s (FDA) consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution considerations for gene therapy products, two industry groups and drugmaker Novartis have suggested changes to the document.
While there are more than 7,000 rare diseases, only two gene therapies have been approved by the US Food and Drug Administration (FDA) to treat inheritable conditions. The Bespoke Gene Therapy Consortium (BGTC) aims to change that.
The National Institutes of Health and Food and Drug Administration are banding together with drugmakers and nonprofits to speed the development of gene therapies for rare diseases. The group is launching the Bespoke Gene Therapy Consortium, with planned funding of $76 million over five years.
A cadre of big and small biotechs and pharmas, plus nonprofit organizations, have signed on to a new U.S. initiative to go after gene therapies for rare diseases. Called the Bespoke Gene Therapy Consortium, or BGTC, the initiative is headed up by the FDA and the National Institutes of Health (NIH) and is designed to accelerate development of gene therapies. The agencies are putting up $76 million over five years.
The National Institutes of Health and U.S. Food and Drug Administration unveiled a public-private partnership Wednesday aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease.
