SpliceBio deal adds extra ‘spark’ to Roche CGT ambitions

Roche subsidiary Spark Therapeutics will use SpliceBio’s protein splicing platform to develop a gene therapy for an undisclosed inherited retinal disease.

Spark Therapeutics will access the ‘Protein Splicing’ technology in a deal that sees it pay Barcelona, Spain-based biotech SpliceBio up to $216 million through upfront, opt-in, and milestone payments.

“This research collaboration and license agreement is an exciting opportunity to develop a novel gene therapy in an area of high unmet medical need,” said SpliceBio CEO Miquel Vila-Perelló. “We are proud that Spark Therapeutics recognizes the potential of our pioneering Protein Splicing platform and the profound impact it could have in the treatment of inherited retinal diseases that are unable to be effectively addressed by other gene therapy approaches.”

DepositPhotos/j.dudzinski

Adeno-associated viruses (AAV) are widely used for the delivery of gene therapies but are limited by their size. They can only package small genes, thus many monogenic diseases cannot be addressed using current technologies.

SpliceBio is addressing this issue through its ‘Protein Splicing’ technology developed in the Muir Lab at Princeton University that uses engineered inteins to catalyze highly efficient protein trans-splicing to reconstitute the desired full-length therapeutic protein in vivo.

Spark Therapeutics is a pioneer in the gene therapy space. The company won US approval in 2017 for Luxturna (voretigene neparvovec), a one-time gene therapy for an inherited retinal disease.

Spark’s $4.3 billion acquisition in 2019 propelled Roche into the advanced therapies space, and the Swiss Pharma Giant has continued to advance its presence through dealmaking. Last year, for example, the firm inked both an AAV capsid deal with Avista Therapeutics and a $110 million allogeneic CAR-T deal with Poseida Therapeutics.