Dive Insight:

BioMarin, a California biotech that built its business around drugs for rare diseases, is a leading gene therapy developer. The Food and Drug Administration reviewed the company’s treatment for hemophilia A last year and, while the agency unexpectedly rejected its application, many expect the regulator to eventually approve the therapy once BioMarin has more follow-up data in hand.

The drugmaker also has gene therapies in earlier stages of testing for a metabolic condition known in shorthand as PKU and a rare disease called hereditary angioedema.

But BioMarin has also been exploring gene therapy for diseases of the central nervous system, although it hasn’t publicly commented on its work in the space until Wednesday’s announcement of the deal with the Allen Institute.

While targeting CNS diseases presents its own challenges, particularly around delivery of treatment, many gene therapy developers are nonetheless working on would-be therapies. According to a count by Cowen, an investment bank, a third of all disclosed preclinical or clinical gene therapy programs as of January 2021 were in neurology, which encompasses more prevalent diseases like Parkinson’s as well as rarer conditions like ALS and Huntington’s.

For BioMarin, the Allen Institute could provide a better tool to target some of these diseases. Adeno-associated viruses are a common delivery tool for gene therapy, but the technology is decades old and has limitations. Several biotech startups, as well as academic laboratories and groups like the Allen Institute, have been researching how to improve the usefulness of AAVs.

Under Wednesday’s deal, scientists from BioMarin and the Allen Institute will work together to evaluate the nonprofit’s modified AAVs for potential use in building more precise CNS-targeted therapies.

“Combining the Allen Institute’s leadership in large-scale genomic science in the central nervous system with BioMarin’s proven experience in developing transformational therapies for rare genetic diseases, lays the foundation to potentially deliver multiple investigational gene therapies to the clinic,” said Lon Cardon, BioMarin’s chief scientific strategy officer, in a statement.

The research behind the Allen Institute’s work on modified AAVs was published last month in the journals Neuron and Cell Reports.