The cell and gene therapy market is forecasted to be worth $81bn by 2029, as per GlobalData’s sales and forecast database. Image Credit: JHVEPhoto / Shutterstock.

Novartis has signed a collaboration and capsid license agreement with Voyager Therapeutics to develop gene therapies for Huntington’s disease and spinal muscular atrophy (SMA).

Voyager will receive $100m in upfront payment, with $20m in equity. The US-based company will also be in line to receive up to $1.2bn in milestone-based payments and tiered royalties on global net sales, as per a 2 January press release.

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Following the news, Voyager stock was up by 24.8% when the market opened on 2 January, compared to the market close on 29 December 2023. The company’s market cap stands at $395.96m today (3 January), with the funds from the current deal expected to extend the company’s runway into mid-2026.

The partnership leverages Voyager’s TRACER technology – tropism redirection of adeno-associated virus (AAV) by cell-type-specific expression of ribonucleic acid (RNA). The TRACER capsid discovery platform allows for the discovery of AAV capsids, which can cross the blood-brain barrier and target the central nervous system while de-targeting the liver and dorsal root ganglia.

The companies have collaborated in the past, with Novartis signing a licensing option agreement with Voyager in March 2022. The agreement allowed Novartis access to Voyager’s technology to develop AAV capsids for use against diseases that affect the central nervous system and the option for two additional targets.

Under the new deal, Novartis will have target-exclusive access to Voyager’s TRACER capsids related to SMA. It will also be responsible for the development and commercialisation of the SMA gene therapy. Novartis’ current portfolio contains an SMA gene therapy, Zolgensma (onasemnogene abeparvovec). The therapy was approved by the US Food and Drug Administration (FDA) for treating type 1 SMA in 2019.

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Following deal closure, Novartis will also gain global rights to Voyager’s AAV gene therapy for Huntington’s. Novartis will bear responsibility for the clinical development and commercialisation of the gene therapy whilst Voyager will be responsible for preclinical advancement.

Novartis has invested in expanding its gene therapy portfolio. In May 2023, it acquired AVROBIO’s haematopoietic stem cell (HSC) gene therapy programme, designed to treat cystinosis. In July 2023, Novartis acquired San Diego-based preclinical stage biotech DTx Pharma for $1bn. DTx Pharma specialises in small interfering RNA (siRNA) therapies for central nervous system and neuromuscular indications.

The cell and gene therapy market is forecasted to be worth $81bn by 2029, as per GlobalData’s sales and forecast database.

GlobalData is the parent company of Pharmaceutical Technology.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.