As it works to rebuild from an earlier gene therapy setback, Astellas is doubling down on the one-and-done modality in a new deal with a London-based startup already in the clinic.
Astellas said Tuesday it will pay $30 million upfront and invest $20 million in AviadoBio in exchange for the chance to exclusively develop and commercialize the startup’s AAV-based gene therapy for people with frontotemporal dementia with progranulin mutations, or FTD-GRN. Investigators dosed the first patient with the experimental medicine, codenamed AVB-101, in April. The Phase 1/2 also opened in the US in July.
Astellas could deliver up to $2.18 billion in license fees and milestone payments, the companies said. Astellas could develop AVB-101 for FTD-GRN and potentially other indications, the duo added. AviadoBio mentions ALS as another focus area for the startup, according to its website.
Patients with FTD, an early form of dementia, can die within three to 13 years after diagnosis, the companies said. Attention, memory, mobility, problem solving and other functions deteriorate in adults with the neurodegenerative disease.
AviadoBio’s gene therapy delivers a working copy of the GRN gene into the brain with the goal of halting the disease’s progress.
The Astellas investment provides a boost to AviadoBio, which does not appear to have raised another financing round since unveiling an $80 million Series A in December 2021.
Lisa DeschampsLeading the upstart is former Novartis Gene Therapies chief business officer Lisa Deschamps.
“Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases,” Deschamps said in a press release, noting the company is completing dosing in the first cohort of the ASPIRE-FTD study.
‘Power of gene therapy’
The AviadoBio tie-up marks a further bet on gene therapy for Astellas, which ran into a major setback in 2020 and 2021, when four boys died after receiving the company’s gene therapy for a rare neuromuscular disease known as X-linked myotubular myopathy.
“People tend to look at the four boys’ deaths. And that’s a very unfortunate and tragic situation,” Astellas CEO Naoki Okamura told Endpoints last month. “On the other hand, there are healthier boys who received [the therapy] and survived for three to five years already. So we believe in the power of gene therapy to really make the difference.”
Astellas is working with Kate Therapeutics to improve the safety of the Audentes gene therapy, which is known as resamirigene bilparvovec, or AT132. The drugmaker also has Phase 1-stage gene therapies for Pompe disease (zocaglusagene nuzaparvovec, or AT845) and ASP2016 for cardiomyopathy associated with Friedreich’s ataxia. It also took a write-down in April on a preclinical Friedreich’s ataxia gene therapy known as AT808 from the Audentes buyout.
Meanwhile, multiple drugmakers are pulling back from gene therapy R&D, including Pfizer, while a small number of startups have been able to reel in large financing rounds to carry forward their work in the field.
The Japanese pharmaceutical company has a gene therapy R&D and manufacturing footprint in the Bay Area; Sanford, NC; and Tsukuba, Japan.