Astellas Bets $50M on Gene Therapy with UK Startup

As Astellas aims to recover from a previous gene therapy setback, it is intensifying its efforts in the one-and-done treatment modality through a new partnership with a London-based startup. On Tuesday, Astellas announced it will pay $30 million upfront and invest an additional $20 million in AviadoBio. This agreement grants Astellas the exclusive rights to develop and commercialize AviadoBio's AAV-based gene therapy for individuals with frontotemporal dementia caused by progranulin mutations (FTD-GRN). The first patient received the experimental treatment, known as AVB-101, in April, and the Phase 1/2 trial commenced in the United States in July.

The deal could potentially result in Astellas providing up to $2.18 billion in license fees and milestone payments. Astellas has the option to develop AVB-101 not just for FTD-GRN but also for other possible indications. AviadoBio has indicated that amyotrophic lateral sclerosis (ALS) is another target area for its therapies.

FTD, a form of early-onset dementia, leads to the deterioration of cognitive functions such as attention, memory, and problem-solving skills. Patients with FTD often have a life expectancy of three to thirteen years post-diagnosis. AviadoBio's gene therapy aims to introduce a functioning copy of the GRN gene into the brain to halt the progression of the disease.

This investment is a significant boost for AviadoBio, which has not reportedly raised additional funds since its $80 million Series A round in December 2021. AviadoBio is led by Lisa Deschamps, former Chief Business Officer of Novartis Gene Therapies. Deschamps expressed optimism about the collaboration, emphasizing the potential to deliver this investigational medicine to families in need of treatment options for FTD-GRN and other neurological conditions.

The collaboration with AviadoBio signifies another commitment to gene therapy for Astellas, despite previous challenges. In 2020 and 2021, Astellas faced a significant setback when four boys died after receiving its gene therapy for X-linked myotubular myopathy, a rare neuromuscular disorder.

Astellas CEO Naoki Okamura addressed the tragedy, highlighting that other patients had survived for several years after treatment. Okamura reaffirmed the company's belief in the transformative potential of gene therapy.

In addition to the AviadoBio deal, Astellas is working with Kate Therapeutics to enhance the safety of its gene therapy for X-linked myotubular myopathy, known as resamirigene bilparvovec or AT132. Astellas is also advancing other gene therapies, including Phase 1-stage treatments for Pompe disease (zocaglusagene nuzaparvovec or AT845) and a therapy for cardiomyopathy related to Friedreich’s ataxia (ASP2016). The company, however, wrote down its investment in a preclinical Friedreich’s ataxia gene therapy (AT808) from the Audentes acquisition.

While several pharmaceutical companies, including Pfizer, are scaling back their gene therapy research and development, Astellas remains committed, supported by its R&D and manufacturing facilities in the Bay Area, Sanford, North Carolina, and Tsukuba, Japan. This commitment contrasts with the trend of reduced investment in gene therapy R&D, as a small number of startups continue to secure substantial financing to advance their projects.