Novartis puts up $1.7bn for Voyager's gene therapy platform

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Voyager Therapeutics has signed another big pharma partner for its TRACER adeno-associated virus (AAV) vector technology, designed to improve the safety of gene therapies, with Novartis taking an option on three neurological disease programmes.

Novartis is paying $54 million upfront to buy in to these three – with another two targets in reserve – and the total value of the deal could reach $1.7 billion including fees and milestones.

Voyager is in line for $37.5 million in fees for each of the first three targets – the identity of which has not been disclosed – if exercised by Novartis.

The deal comes after Pfizer agreed a $630 million deal with Voyager last October, with $30 million upfront, focusing on gene therapies for neurological and cardiovascular diseases, and helps to reverse a trend in which three other former partners bailed on partnerships with the biotech.

The deal is further evidence of a renaissance at Voyager, which lost three other gene therapy partners – Sanofi, AbbVie and Neurocrine Biosciences – in 2019 and 2020. It also provides financial stability, as according to the company it provides funding into 2024.

The increased financial muscle afforded by the new deal will be welcomed by Al Sandrock, Biogen's former head of R&D who was appointed to Voyager's board and executive committee earlier this year.

TRACER capsids are designed to target cells and tissues in the body more effectively, allowing the dose of gene therapy to be reduced and hopefully fewer side effects than conventional AAV-delivered therapies.

Renewed interest in Voyager's technology platform has almost certainly been sparked by a string of safety scares in the last couple of years involving gene therapies using other AAV vector technologies.

Voyager's in-house gene therapy development efforts have had some issues too, for example its VY-HTT01 gene therapy for Huntington disease was placed on clinical hold by the FDA at the end of 2020, which has since been relaxed although the candidate has been shelved.

Voyager is still working on Huntington's, as well as other gene replacement therapies for spinal muscular atrophy, diseases linked to GBA1 mutations(including Parkinson's disease, Lewy body dementia, and Gaucher disease.

A parallel effort is using TRACER capsids to deliver antibodies for HER2-positive cancers as well as Alzheimer's disease.

Novartis said it intends to use TRACER to "help deliver therapies to previously intractable regions of the brain, potentially overcoming barriers that have hindered development of gene therapies for neurological diseases to date."