SpliceBio Enters Collaboration with Spark Therapeutics to Develop aGene Therapy Targeting an Inherited Retinal Disease•SpliceBio is eligible to receive upfront, opt-in and milestone payments up to $216 million
plus royalties•Spark secures exclusive worldwide rights to SpliceBio’s proprietary Protein Splicing
platform to develop, manufacture, and commercialize a gene therapy targeting anundisclosed inherited retinal disease

BARCELONA, 17 October, 2023 – SpliceBio, a genetic medicines company harnessing
Protein Splicing to develop the next generation of gene therapies, is pleased to announce thesigning of an exclusive collaboration and licensing agreement with Spark Therapeutics toutilize SpliceBio’s proprietary Protein Splicing platform to develop a gene therapy for an
undisclosed inherited retinal disease.
Under the terms of the agreement, SpliceBio and Spark will conduct a research collaboration
utilizing SpliceBio’s proprietary Protein Splicing platform, which offers the potential to address
diseases that currently cannot be treated with gene therapies because the necessary gene is
too large to be delivered by adeno-associated virus (AAV) vectors. Spark will have exclusive
worldwide rights to develop, manufacture, and commercialize a gene therapy arising from thisresearch collaboration targeting an undisclosed inherited retinal disease. SpliceBio will beeligible to receive upfront, opt-in and milestone payments up to $216 million and royalties on
net sales.
“This research collaboration and license agreement is an exciting opportunity to develop anovel gene therapy in an area of high unmet medical need. We are proud that Spark
Therapeutics recognizes the potential of our pioneering Protein Splicing platform and theprofound impact it could have in the treatment of inherited retinal diseases that are unable tobe effectively addressed by other gene therapy approaches,” said, Miquel Vila-Perelló,Ph.D., Chief Executive Officer and co-founder of SpliceBio. “In addition to the Sparkcollaboration, we continue to develop our lead program in Stargardt disease and further build
our capabilities and pipeline of wholly-owned gene therapy programs to develop life-changing
therapies for patients in need.”“This partnership builds on Spark’s leadership in gene therapies for inherited retinal diseases.
Our breakthrough gene therapy LUXTURNA® demonstrated how we can change the lives ofpatients with biallelic mutations in the RPE65 gene whose physicians have determined theireligibility for treatment. At the same time, there are many more people with other inherited
retinal diseases that need treatment options,” said Federico Mingozzi, PhD, Chief Science &Technology Officer of Spark Therapeutics. “With our complementary capabilities, combined
deep technical knowledge and SpliceBio’s impressive protein splicing platform we hope tomake further progress in the treatment of inherited retinal diseases, bringing new genetherapies into the clinic and to commercial availability.”-Ends-

About SpliceBio
SpliceBio is a genetic medicines company harnessing its proprietary Protein Splicing platform
to develop the next generation of gene therapies. The Company’s platform offers the potential
to address diseases that currently cannot be treated with gene therapies because thenecessary gene is too large to be delivered by adeno-associated virus (AAV) vectors. The
company’s lead program targets Stargardt disease, a genetic eye disease that causes vision
loss in children and adults. SpliceBio’s platform is based on technology developed in the Muir
Lab at Princeton University after more than 20 years of pioneering intein and protein
engineering research. For additional information, please visit www.splice.bio.
About Spark Therapeutics
Spark Therapeutics is a fully integrated, commercial company dedicated to unlocking thepower of gene therapy to accelerate healthcare transformation. At Spark, a member of theRoche Group, we see the path to a world where no life is limited by disease. For more
information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.
For further information:
Optimum Strategic Communications
Mary Clark, Zoe Bolt, Eleanor Cooper
Email: splicebio@optimumcomms.com
Tel: +44 (0) 20 3882 9621
Spark Therapeutics
Denise Bradley
Email: communications@sparktx.com