While U.S. lawmakers continue their debate on reducing spending for prescription drugs, government payers are exploring innovative reimbursement ideas to cover gene and cell therapies that could cost millions of dollars for a cure or a durable effect against rare diseases.
Biogen Inc. has announced contrasting results from phase III trials of therapies for a rare eye disease and depression, following last week’s controversial FDA approval of Alzheimer’s drug Aduhelm. The Cambridge, Mass.-based firm said a phase III gene therapy study in the rare retinal disease choroideremia missed its primary and secondary endpoints, although the news was better from a potential therapy for major depressive disorder.
LONDON – The pioneers of gene therapy in Europe now aim to take the field to the next level, moving into the development of virally delivered antibodies in the treatment of central nervous system and muscular diseases, with Alzheimer’s disease one of the first targets.
The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
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.
RSS
