Regeneron Pharmaceuticals and Mammoth Biosciences have partnered to research, develop and commercialise in vivo CRISPR-based gene editing therapies for multiple tissues and cell types.

US-based Regeneron is developing adeno-associated viral vectors (AAVs) that utilise antibody-based targeting methods to deliver genetic medicines to specific tissues and cells.

Mammoth Biosciences is a biotechnology company working on ultracompact nucleases and related gene editing systems. These systems offer a range of editing capabilities compared to traditional CRISPR-based systems.

The collaboration will combine Regeneron`s expertise in AAVs and antibodies with Mammoth`s knowledge in compact gene editing. Both entities will develop treatments for diseases that affect tissues beyond the liver.

Regeneron senior vice president and Regeneron Genetic Medicines co-head Christos Kyratsous said: “We believe in the incredible power of gene editing, which we are utilizing in our diverse preclinical and clinical genetic medicines pipeline.

“After years spent developing our next-generation delivery approaches, we are eager to combine them with Mammoth’s gene editing systems to better match payload, delivery system and disease type.”

Under the agreement, Mammoth will initially receive $100m, which includes $95m in equity investment upon signing, along with an upfront payment.

Mammoth stands to gain up to $370m per target via various milestone payments during development, regulatory approvals, and commercialisation.

The company will also receive royalties ranging from single digits to mid-teens on future net sales of collaborative products.

Additionally, the biotechnology company retains the option to participate in co-funding and profit-sharing arrangements for most collaboration projects instead of receiving milestone payments and royalties.

In return, Regeneron Pharmaceuticals gains broad access to Mammoth`s editing technologies, excluding certain targets, for five and a half years. This access may be extended for an additional two years upon payment of a research extension fee.

Both parties will collaborate in selecting and researching targets, with Regeneron leading the development and commercialisation activities.

Mammoth Biosciences co-founder and CEO Trevor Martin said: “Mammoth’s ultracompact CRISPR systems address the size constraints of viral delivery and complement Regeneron’s targeted AAV technologies.

“We look forward to working with Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing.”