Gene Therapies

Novartis announced its partnership with Voyager Therapeutics to advance the development of gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA), harnessing Voyager’s innovative adeno-associated virus (AAV) capsid discovery platform. This collaboration could potentially escalate Voyager’s proceeds from the alliance to an estimated $2 billion.

The centerpiece of this collaboration is Voyager’s Tropism Redirection of AAV by Cell-type-specific Expression of RNA (TRACER™) technology. This RNA-based platform is designed for rapid discovery of AAV capsids that effectively penetrate the blood-brain barrier and demonstrate enhanced central nervous system (CNS) tropism. In preclinical studies, TRACER-generated capsids have shown promising results, offering widespread gene expression in the CNS, targeting difficult-to-reach brain areas, and minimizing impact on the liver and dorsal root ganglia.

Under the agreement, Voyager will grant Novartis exclusive access to its TRACER capsids for SMA, overseeing all development and commercialization processes. Additionally, Novartis will acquire worldwide rights to Voyager’s AAV gene therapy for HD, including the TRACER capsids and proprietary payloads. For the HD program, Voyager will lead preclinical development while Novartis will handle clinical development and commercialization.

Financially, Novartis commits to an $80 million upfront payment to Voyager and a $20 million investment in Voyager stock. The deal also includes development, regulatory, and commercialization milestone payments up to $200 million for the SMA program and $225 million for the HD program, along with significant sales milestone payments and royalties.

The announcement has already influenced the market, with Voyager shares surging nearly 30%, reflecting investor confidence in this expanded collaboration. In contrast, Novartis shares showed a modest increase.

The partnership, initiated in March 2022, initially focused on developing gene therapies for up to three undisclosed neurological diseases. Since then, the agreement has evolved, with Novartis exercising options for novel TRACER-generated capsids and Voyager entering into collaborations with several other biopharma partners.

Novartis and Voyager express strong optimism about this collaboration. Fiona Marshall, president of Biomedical Research at Novartis, emphasizes the potential of TRACER capsids in advancing gene therapies for CNS diseases. Voyager CEO Alfred W. Sandrock, Jr., MD, PhD, highlights the significance of this partnership in advancing new therapies for patients and strengthening Voyager’s financial stability.

This partnership marks a significant step in the development of gene therapies, offering hope for patients with severe neurological conditions and illustrating the potential of collaborative innovation in the biotech sector.

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