Two Very Different Flavors Of Ocular Gene Therapy Get A Boost
The 2021 collaboration between Abbvie and Regenxbio to develop an anti-VEGF gene therapy for chronic retinal diseases is a noteworthy deal in the gene therapy space. But for both clinical and commercial reasons, it is a one-off opportunity and not an indicator of the overall potential of gene therapies to address eye diseases. Interest in optogenetics, however, which is the introduction of light-sensing genes into cells in the eye to restore vison, may be on the rise.
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The partnership worth up to $1.75bn removes the significant risk of going it alone in a competitive market as Regenxbio awaits late-stage data for its gene therapy.
Higher success rates in rare cancers, where biomarkers can better direct therapy, characterize the oncology treatment landscape. Deal-making is favoring alliances over acquisitions as drugmakers focus on establishing the clinical benefit of new targets, particularly for rare indications.
Brazil has established an accelerated pathway for approval of drugs for ultra-rare diseases, encouraging development activity – but the definition of qualifying drugs is narrow. Expansion to include more treatments could boost clinical trials activity and companies’ overall development presence.