Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
In the largest collaboration of 2024, Sarepta Therapeutics Inc. and Arrowhead Pharmaceuticals Inc. entered a sprawling global licensing deal that includes a swath of clinical and preclinical candidates targeting rare genetic diseases. Under the terms, Sarepta gains access to existing and potential future compounds derived from the RNAi platform developed by Arrowhead, with the latter eligible for payments potentially exceeding $11 billion.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
The Ministry of Food and Drug Safety (MFDS) approved South Korea’s first denosumab (Prolia/Xgeva) biosimilars, developed by Celltrion Inc. under the brand names of Stoboclo/Osenvelt (CT-P41) for respective indications, a move the company hopes will help secure first-mover advantage for the drugs, currently under review in the U.S. and Europe.
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
As an array of developers push their Friedreich’s ataxia (FA) drugs along, PTC Therapeutics Inc.’s recent update on its program piqued thirst for the 15-lipoxygenase inhibitor vatiquinone. Warren, N.J.-based PTC said the prespecified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
It’s a go for the two phase III Ensure studies of Immunic Inc.’s lead asset in treating relapsing multiple sclerosis. An unblinded data monitoring committee’s interim futility analysis concluded that the placebo-controlled, pivotal studies using vidofludimus calcium may continue as planned, with the program expected by the company to be completed in 2026.
The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
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