China is making strides in cell and gene therapy, notably so with a 61% surge in the number of clinical trials in six years, a new report by Ernst & Young showed. While analysts noted the increasing innovation efforts and cross-border collaborations, concerns remain if quality will be compromised by speed.
Less than a year after Novartis AG's acquisition of optogenetics specialist Vedere Bio Inc., its successor Vedere Bio II Inc. is launching with $77 million in series A financing, led by Octagon Capital. The company will develop earlier-stage assets than those Novartis purchased, including new, mutation-agnostic optogenetics technology to improve upon current gene therapies aimed at restoring functional vision to people with vision loss due to photoreceptor death.
Little more than a year ago, when COVID-19 lockdowns began, turned out to be a prime time for finding the right funding and partners. That’s when privately held Capsigen Inc. was on a search that ended with a Biogen Inc. deal that could add up to $1.3 billion.
The allocation of capital to the build-out of next-generation gene therapies continues apace. Dyno Therapeutics Inc., a leader in applying artificial intelligence to advanced capsid engineering, raised $100 million in a series A round to fund its expansion and that of its Capsidmap platform.
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.
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