The two deals will provide Vertex with investigational gene therapies for Duchenne muscular dystrophy (DMD) and mytonic dystrophy type 1 (DM1).
The acquisition of Exonics Therapeutics was agreed after Vertex offered to pay $245m (€216m) upfront for the company, with potential future payments rounding this figure up to an approximate $1bn.
Exonics’ research is still at the pre-clinical stage, focusing on DMD by using adeno-associated virus (AAV) to repair exon mutations and restore the production of dystrophin – the absence of which causes DMD and results in progressive muscle degeneration.
Vertex’s decision to expand its existing collaboration and licensing agreement with CRISPR Therapeutics also potentially sets the latter company up for a $1bn windfall. It will receive $175m upfront, with potential further payments making up the rest of the figure.
Vertex will take the lead on the R&D, manufacture and commercialization for CRISPR’s DMD program. The two companies are already partnered on the clinical development of CTX001, a stem cell treatment for haemoglobinopathies.
“Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform for DMD and DM1,” said Jeffrey Leiden, CEO of Vertex.
Both deals are expected to complete in the third quarter of 2019.
In another related addition, Vertex stated that it will appoint John Gray as SVP of genetic therapies at the company, effective as of June 17.