The development of gene therapy has come a long way over the past two decades after getting off to a rocky start following the death of a young patient after being treated with an experimental therapy. Since that time continuing scientific progress has enabled the development of a robust product pipeline of promising therapies that could lead to, according to FDA estimates, 10 to 20 cell and gene therapy products a year within the next five years. The renaissance of the sector has also attracted record amounts of investment capital and significant business development.
Cambridge, Mass.-based Agios Therapeutics Inc.’s encouraging phase III data from a pair of trials with allosteric activator mitapivat in pyruvate kinase deficiency brought more attention to the space, where Rocket Pharmaceuticals Inc. – at a much earlier stage – is trying a gene therapy called RP-L301.
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.
PARIS – Whitelab Genomics SAS has signed a partnership agreement with Genethon, a French research center in the field of gene therapy, to ramp up development of genetic therapy techniques using artificial intelligence (AI). As part of this partnership, Genethon will use the Whitelab Genomics Catalyst platform to develop new capsids.
Elevatebio LLC, a provider of infrastructure, expertise and investment for a growing roster of cell and gene therapy companies, has raised $525 million in series C financing to advance its work. Matrix Capital Management led the round, joined by new investors Softbank Vision Fund 2 and Fidelity Management and Research Co. Existing investors also joined in.
Tenaya Therapeutics Inc. raised $106 million in a series C round to continue its build-out as a fully integrated gene therapy and regenerative medicine specialist focused on cardiovascular disease.
Jaguar Gene Therapy LLC, a startup reuniting former Avexis Inc. executives to develop a portfolio of potential treatments for severe genetic diseases, announced its public debut Feb. 25 with more than $40 million in series A financing from co-creator Deerfield Management.
Avrobio Inc. said Feb. 8 that the first person dosed with AVR-RD-01, an investigational ex vivo lentiviral gene therapy from its upgraded manufacturing platform, Plato, experienced a complete clearance of the toxic substrate lyso-Gb3 in a kidney biopsy.
While it’s too late to save the contingent value rights connected with the acquisition of Celgene Inc., Bristol Myers Squibb Co.’s CD19-targeted CAR T therapy, lisocabtagene maraleucel, for treating certain types of relapsed or refractory large B-cell lymphoma patients who have received at least two prior therapies, won FDA approval.
At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.
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