June 18 (Reuters) - Swiss drugmaker Roche will
team up with privately held biotech Ascidian Therapeutics to
develop gene therapies targeting difficult to treat neurological
diseases, the Boston-based startup said on Tuesday.
WHY IT'S IMPORTANT
Roche is pursuing several therapeutic fields to offset
falling oncology sales, setting a high deal pace to restore its
development pipeline that was hit by major trial setbacks in
Alzheimer's and cancer immunotherapy in 2022.
Under the agreement, Roche gets exclusive rights to
Ascidian's RNA exon editing technology to develop therapies for
undisclosed neurological diseases.
BY THE NUMBERS
Ascidian will receive an initial payment of $42 million and
up to $1.8 billion in research, clinical, and commercial
milestone payments. It is also eligible to receive royalties on
commercial sales worldwide for any therapies developed under the
partnership.
CONTEXT
Ascidian, backed by venture capital firm Apple Tree
Partners, is separately testing a genetic eye disease therapy in
an early-stage trial. It raised $40 million in its latest
funding round in November 2023.
The therapy candidate, ACDN-01, is also based on its RNA
exon editing technology that involves replacing mutated sections
of genes with healthy ones.
This approach aims to provide long-lasting effect similar to
traditional gene therapy but with reduced risks compared to
direct DNA editing and gene replacement.
KEY QUOTES
"Our partnership with Ascidian is an opportunity to harness
advanced RNA exon editing technology, which has the potential to
deliver transformative one-time therapeutics by editing
multiple whole exons at the RNA level with a single treatment,"
James Sabry, global head of pharma partnering at Roche, said.