Taysha buys clinic-ready gene therapy from Abeona, bringing Steven Gray's portfolio back in one place
Less than four months after Taysha launched with a suite of AAV gene therapies developed at UT Southwestern — and two weeks after the ex-AveXis team raised $95 million in fresh cash — the biotech is looking to its chief scientific advisor for another asset.
The new program is ABO-202, which is designed to treat CLN1 disease (or infantile Batten disease) by delivering a correct copy of the PPT1 gene with a modified AAV9 vector.
Steven Gray developed the therapy while at UNC-Chapel Hill, where he had studied under Jude Samulski years ago. Abeona licensed it in 2016, built out the clinical trial protocol and manufacturing process, and has since had an IND for a Phase I/II clinical trial cleared with the FDA.
Taysha is paying only $7 million upfront to get its hands on ABO-202, with $3 million in license fees and $4 million for the inventory — including clinical-grade CLN1 plasmid. Abeona is eligible to receive up to $56 million down the road for development and sales milestones, plus royalties.
The deal puts Taysha on track to begin two clinical studies next year: both the CLN1 program and another gene therapy for Tay-Sachs disease. Three more INDs are on the docket for 2021.
“CLN1 is a progressive monogenic CNS disease with significant unmet medical need, and we believe the ABO-202 data generated thus far demonstrate great translational potential and offer hope to children suffering from this devastating disorder,” CEO RA Session II said.
Gray, who now leads 50 translational scientists and manages a GMP manufacturing site at UT Southwestern’s viral vector core, will continue to advise the company alongside Berge Minassian, the division chief of child neurology.
Rather than shepherding one asset at a time like the first generation of gene therapy players, Taysha had opted for a portfolio approach to advance multiple gene therapies in parallel. It’s looking to build three big franchises: genetic forms of epilepsy, neurodevelopmental disorders and neurodegenerative disorders. And although the crew has eyes on newer technologies like redosing, bicistronic plasmids, microRNA knockdown, hairpin technology to turn on a silent allele and delivery through the vagus nerve, the cornerstone remains adeno-associated virus (AAV).
“If it’s not broke, you don’t fix it,” Session told Endpoints News back in April.
Taysha, which is chaired by former AveXis CEO Sean Nolan, has reserved rights to tap four more programs out of Gray’s lab.
For Abeona, the deal with Taysha gives them time and some cash to focus on its more advanced programs in recessive dystrophic epidermolysis bullosa, Sanfilippo syndrome type A and Sanfilippo syndrome type B as it recovers from a somewhat tumultuous 2019.