April 25 (Reuters) - Regeneron Pharmaceuticals ( REGN )
and Mammoth Biosciences will collaborate to research, develop
and commercialize gene editing therapies for multiple diseases,
the companies said on Thursday.
Mammoth's CRISPR-based gene editing platform and Regeneron's
delivery technologies will be used to create disease-modifying
medicines that can be delivered to tissues beyond the liver.
CRISPR, discovered by Jennifer Doudna and CRISPR
Therapeutics co-founder Emmanuelle Charpentier, uses
molecular "scissors" to trim faulty parts of genes that can then
be disabled or replaced with new strands of normal DNA.
In 2023, the U.S. FDA approved five gene therapies,
including a sickle cell disease treatment from Vertex
Pharmaceuticals ( VRTX ) and CRISPR Therapeutics that uses the
latter's gene editing technology.
Regeneron joins drugmakers Bayer and Vertex
Pharmaceuticals ( VRTX ) in striking partnership deals with
Mammoth for development of gene therapies.
Mammoth, which was founded by Doudna, will receive $100
million in total upfront payment and equity investment from
Regeneron and could receive up to $370 million per therapy in
development, regulatory and commercial milestone payments.
The parties will jointly select and research for the
therapies, and then Regeneron will lead development and
commercialization.