RESEARCH TRIANGLE PARK – AskBio, a gene therapy company based in RTP that recently raised $225 million from investors, will pay up to $240 million in upfront and milestone payments for a license to use gene therapy technology from Massachusetts-based Selecta Biosciences as part of a regime to treat Pompe disease.
The license is for ImmTOR, what Selecta describes as a immune tolerance platform.” The technology is “addressing barriers to repeat administration” of gene therapies.
Pompe disease affects between 5,000 and 10,000 people a year, affecting “ventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills,” AskBio says.
“There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selecta’s ImmTOR technology,” said Sheila Mikhail, CEO and co-founder of AskBio, in a statement. “The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.”
The companies initially announced a partnership in August.