Pioneering Protein Splicing:
A Novel Gene Therapy Modality

SpliceBio is a genetic medicines company harnessing its proprietary Protein Splicing platform to develop the next generation of gene therapies. Our 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.

SpliceBio Announces First Patient Dosed in Phase 1/2 ASTRA Study of SB-007, a Dual-AAV Gene Therapy for Stargardt Disease

BARCELONA, SPAIN, 13 March 2025 – SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced that it has dosed the first patient in the Phase 1/2 ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration, leading to progressive central vision loss and ultimately resulting in blindness, with no approved treatments available.

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Our Team

CO-FOUNDER & CEO

Miquel Vila-Perelló, Ph.D

Miquel co-founded SpliceBio and serves as CEO. Miquel has broad experience in biotechnology, chemical biology and protein chemistry as well as in the managing of research teams and projects. After obtaining his PhD at the University of Barcelona he carried out postdoctoral studies at The Rockefeller University. He later became a Research Scholar at Princeton University, where he managed the Protein Center at the Department of Chemistry. Over the last ten years he has contributed to the discovery and development of novel protein ligation technologies. He has authored more than 23 publications, including patent applications, and has a proven track record for the leadership of scientific teams and non-dilutive fund raising.

CO-FOUNDER & CTO

Silvia Frutos, Ph.D

Silvia co-founded SpliceBio and serves as CTO. Silvia has broad experience in chemical biology, protein chemistry as well as peptide synthesis. She carried out her PhD at the Barcelona Science Park. After that she moved to New York to carry out her postdoctoral studies at The Rockefeller University. Over the last ten years she has contributed to understand the mechanism of the inteins, which are essential in our protein ligation technology. Also Silvia brings deep scientific expertise and an intimate knowledge of European bioclusters.

CHIEF BUSINESS OFFICER

Gerard Caelles

Gerard joined SpliceBio in 2016 as the responsible for Business Development operations. Prior to joining SpliceBio, Gerard served as the Business Development Manager at Bionure, where he was focused on business development and partnering around the company’s novel candidate in the orphan neuro-ophthalmology space, as well as on fundraising efforts. Gerard holds a BSc in Biotechnology from Universitat Autònoma de Barcelona and has been studying Mandarin Chinese for four years.

CHIEF MEDICAL OFFICER

Aniz Girach, MD

Aniz joined in 2024 and serves as Chief Medical Officer.
Dr Girach is an ophthalmologist with over 25 years’ industry experience in the field of Medical Retina and Genetic Therapy for inherited retinal diseases. He has been involved in the development and approval of four drugs in ophthalmology. Dr Girach was Global Head of Ophthalmology at Merck & Co and Vice President, Clinical Development at Alcon Laboratories. At Oxurion NV (formerly ThromboGenics) he was Global Head of Ophthalmology and Chief Medical Officer where he oversaw the development and approval of Ocriplasmin (Jetrea), a first in class biologic therapy for retinal disease. Dr Girach was Chief Medical Officer at Nightstar Therapeutics where he led and oversaw the development of its gene therapy programs for inherited retinal diseases, prior to its acquisition by Biogen for $800 million. Prior to joining SpliceBio, he led the development of genetic therapies for inherited retinal diseases at ProQR Therapeutics NV as its Chief Medical Officer.

Dr Girach spent 11 years in Clinical Practice and Academia before joining the pharmaceutical industry with Eli Lilly as its Senior Global Ophthalmologist and Medical Director where he specialized in retinal diseases. He is a Visiting and Honorary Professor at Wills Eye Hospital, Philadelphia, USA, and is a reviewer for five peer-reviewed international ophthalmology journals. He has published four textbooks in ophthalmology and has published over 90 abstracts and manuscripts in peer-reviewed journals in ophthalmology, with numerous invited lectures at national and international ophthalmology meetings. He currently holds Independent Board of Directorship positions for two biotechnology companies and is also the Chair of the R&D Committees for both companies.