Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
Through a $600 million deal, Juniper Biologics Pte. Ltd. has acquired rights from Kolon Life Science Inc. to develop and commercialize TG-C LD (Tissuegene-C low dose) for the treatment of knee osteoarthritis. Singapore-based Juniper will hold the rights for Asia Pacific, the Middle East and Africa and be responsible for developing and commercializing the candidate within those regions.
Japan’s Astellas Pharma Inc. is continuing its investment in gene therapies, following up its $3 billion acquisition of Audentes Therapeutics Inc. with a technology licensing deal with Dyno Therapeutics Inc. potentially worth more than $1.6 billion. Central to the deal is Cambridge, Mass.-based Dyno’s adeno-associated virus (AAV) vector technology, which can be used to direct gene therapies to skeletal and cardiac muscle.
Poseida Therapeutics Inc.’s R&D Day in February – where much of its technology was made public for the first time – created “a flood of interest” in deals and officials were “pretty selective,” said CEO Eric Ostertag, whose remarks came as the company nailed down a whopping research collaboration and exclusive license agreement with Takeda Pharmaceutical Co. Ltd. The arrangement will deploy Poseida’s Piggybac and Cas-CLOVER, as well as biodegradable DNA and RNA nanoparticle delivery technology and other genetic engineering platforms to come up with as many as eight gene therapies.
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.
Astellas Pharma Inc. has halted further dosing of the experimental X-linked myotubular myopathy (XLMTM) gene therapy AT-132 (resamirigene bilparvovec) after one participant in the ongoing Aspiro study experienced a serious adverse event (SAE) of abnormal liver function.
China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
Cell and gene therapy have seen much progress in recent times, with the product pipelines in those areas bursting with more than 1,200 therapies. Naturally, the challenges, opportunities and essential development strategies in those fields were the focus on the last day of the BIO Asia-Taiwan Conference 2021.
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