SpliceBio and Spark Therapeutics, Inc. have agreed to an exclusive collaboration and licensing agreement to develop a gene therapy for an inherited retinal disease (IRD).
Let’s start with a refresher.
Philadelphia, Pennsylvania-headquartered Spark Therapeutics, a subsidiary of the Roche Group, is a gene therapy company focused on developing treatments for genetic diseases.
Its FDA-approved gene therapy Luxturna (voretigene neparvovec-rzyl) is an adeno-associated virus (AAV) vector-based gene therapy which is indicated for treating patients with biallelic RPE65 mutation-associated retinal dystrophy.
See the prescribing info here.
Now SpliceBio.
Based in Barcelona, Spain, SpliceBio is a genetic medicine company targeting the development of next-generation gene therapies to treat genetic diseases via a proprietary platform (more on that below). The company’s lead program is focused on Stargardt disease.
Talk about this proprietary platform.
First off: “Protein splicing” is a multi-step biochemical reaction that leads to the formation of peptide bonds. This process is carried out by a family of auto-processing-domain proteins called “inteins”.
A next-gen version of these proteins are currently in development for therapeutics use: engineered inteins, which are at the center of SpliceBio’s Protein Splicing platform.
The platform was developed with, “the potential to address diseases that currently cannot be treated with gene therapy because the necessary gene is too large to be delivered by adeno-associated virus (AAV) vectors.”
Gotcha. Now this partnership.
Per the agreement, both companies will collaboratively conduct research using the Protein Splicing platform. Spark will be granted the exclusive global rights to develop, manufacture, and commercialize a gene therapy based on this research to target a (currently undisclosed) IRD.
Any financials disclosed?
Yup; SpliceBio is eligible to receive upfront, opt-in, milestone payments of up to $216 million.
Lastly .. didn’t Spark just announce another collaboration?
A while back, in January 2023. The company partnered with Neurochase Ltd. to develop gene therapies for rare central nervous system (CNS) disorders, with a focus on delivery mechanisms.