Voyager Therapeutics Inc. is getting $30 million up front in a potential $630 million gene therapy deal with Pfizer Inc., the company’s first such agreement since a strategic refocusing effort earlier this year and a much-needed endorsement of a next-generation AAV capsid platform that has shown promising though early stage data.
Selecta Biosciences Inc. joined a licensing agreement with Takeda Pharmaceutical Co. Ltd. to develop AAV-driven gene therapies for two lysosomal storage disorders that could bring Watertown, Mass.-based Selecta up to $1.124 billion. The payments depend upon hitting development or commercial milestones. Selecta is also receiving an undisclosed up-front payment and is eligible for tiered royalties on commercial sales.
Selecta Biosciences Inc. joined a licensing agreement with Takeda Pharmaceutical Co. Ltd. to develop AAV-driven gene therapies for two lysosomal storage disorders that could bring Watertown, Mass.-based Selecta up to $1.124 billion. The payments depend upon hitting development or commercial milestones. Selecta is also receiving an undisclosed up-front payment and is eligible for tiered royalties on commercial sales.
As if it were needed, Amicus Therapeutics Inc.’s spin-off of its gene therapy work and PDUFA VII’s provisions to increase the capacity of the FDA’s Center for Biologics Evaluation and Research offered further proof this week of the global explosion that’s happening in the regenerative medicine field.
Amicus Therapeutics Inc.’s plan to spin off its gene therapy work by way of the combination with blank check firm Arya Sciences Acquisition Corp. IV “allows us to be laser-focused on maximizing the opportunity for Galafold [migalastat]” while setting up launch preparations for AT-GAA, said Bradley Campbell, chief operating officer.
The PDUFA commitment letter negotiated between industry and the U.S FDA every five years provides an inside look at the future of drug development. The PDUFA VII letter, which is to be presented to Congress by Jan. 15, is no exception.
LONDON – There were well-deserved celebrations in Paris on Sept. 20, as Jeito Capital toasted the oversubscribed close of its first fund at €534 million (US$625.5 million). This is claimed as the largest European venture fund dedicated to life sciences, exceeding the original target of €500 million, and with €340 million of the total raised under the constraints of the pandemic from January 2020 onward.
LONDON – Anjarium Biosciences AG has raised $61 million in a series A to advance development of a new class of gene therapy it is developing from the ground up to overcome shortcomings of current viral vector-based products. The Zurich, Switzerland-based company is assembling a toolkit of stable DNA vectors and targeted lipid nanoparticle and exosome delivery vehicles that it said will lead to “bespoke expression of genes” and address the underlying causes of genetic diseases in a more predictable, repeatable and lasting way.
A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
Abbvie Inc. and Regenxbio Inc. have announced a partnership to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy and other chronic retinal diseases. Under the terms of the agreement, Abbvie will pay Regenxbio $370 million up front, plus up to $1.38 billion in additional development, regulatory and commercial milestones. The deal gives '314 – already the most advanced gene therapy in wet AMD – another potential edge against its nearest competitor, Adverum Biotechnologies Inc.’s ADVM-022.
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