Pioneering Protein Splicing:
A Novel Gene Therapy Modality

Dr. Miquel Vila-Perelló is the co-founder and Chief Executive Officer of SpliceBio, a genetic medicines company he has led since its inception in 2014 (formerly ProteoDesign). Under his leadership, SpliceBio has grown from an early-stage concept into a global clinical-stage biotechnology company, advancing multiple programs into development—including the lead program for Stargardt disease currently in the clinic—and securing over $200 million in funding from top-tier investors.

The company’s foundation and technology are rooted in Dr. Vila-Perelló’s pioneering research on protein splicing and engineering at Princeton University, where he served as a Research Scholar and managed the Protein Center in the Department of Chemistry. Prior to that, he spent six years at The Rockefeller University as a postdoctoral researcher, contributing to the development of innovative protein ligation technologies.

Dr. Vila-Perelló has dedicated the past two decades to advancing protein engineering for therapeutic development, both as a scientist and as a biotech executive. As CEO of SpliceBio, he has overseen the company’s scientific and corporate growth, from early research to clinical stage and international expansion. He is also the author of over 23 peer-reviewed publications and the inventor of multiple granted patents. Dr. Vila-Perelló holds a PhD in Chemistry from the University of Barcelona.

Dr. Silvia Frutos is the Co-Founder and Chief Technology Officer of SpliceBio, where she leads the development of the company’s core technology platform and scientific strategy. She brings over two decades of experience in chemical biology, protein chemistry, and peptide synthesis, and has been instrumental in advancing SpliceBio’s Protein Splicing platform for gene therapy applications.

Dr. Frutos’s deep expertise in inteins and protein splicing has been critical to the company’s innovation efforts, enabling the design of novel technologies for precise and efficient delivery of genetic medicines. She plays a central role in translating cutting-edge science into therapeutic solutions, guiding the company’s research, technical teams, and platform evolution.

Before co-founding SpliceBio, Dr. Frutos was a CIBER researcher at the University of Barcelona, where she investigated the molecular dynamics of DNA interactions. Earlier, she completed postdoctoral training at The Rockefeller University, where she contributed to foundational work on intein-mediated protein splicing mechanisms. Dr. Frutos holds a PhD in Chemistry from the University of Barcelona.

Mr. Gerard Caelles is the Chief Business Officer at SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes. Since joining SpliceBio in 2016, he has played a pivotal role in the company’s growth from a platform company to a gene therapy-focused company, driving its seed, $57M Series A and $135M series B financings and forging strategic partnerships with key stakeholders in the biopharmaceutical industry. Beyond corporate and business strategy, he has been instrumental in shaping SpliceBio’s pipeline, playing a key role in defining the company’s R&D strategy. He is also a co-inventor of several of the company’s core technologies.

Gerard has dedicated his career to building biotechnology startups focused on rare diseases. Before SpliceBio, he led business development at Bionure and contributed to the development of BN-201, now OCS-05, a novel drug in Phase 2 trials for a rare neuro-ophthalmology condition called Acute Optic Neuritis. Gerard holds a degree in Biotechnology from the Universitat Autònoma de Barcelona. A biotechnologist at heart, his life is driven by the ambition to turn cutting-edge science into meaningful therapies for patients.

Dr. Aniz Girach joined SpliceBio in 2024 as the Chief Medical Officer, bringing over 25 years of experience in medical retina and gene therapy for inherited retinal diseases. His extensive background includes involvement in the development and approval of four ophthalmology drugs. Prior to SpliceBio, Dr. Girach held key positions such as Global Head of Ophthalmology at Merck & Co, Vice President of Clinical Development at Alcon Laboratories, and Chief Medical Officer positions at Oxurion NV(ThromboGenics), Nightstar Therapeutics, where he led gene therapy programs for inherited retinal diseases throughout clinical development culminating in the company’s acquisition by Biogen for $800 million, and most recently at ProQR Therapeutics.

Dr. Girach spent 11 years in clinical practice and academia before transitioning to the pharmaceutical industry with Eli Lilly as Senior Global Ophthalmologist and Medical Director, specializing in retinal diseases. He is a Visiting and Honorary Professor at Wills Eye Hospital in Philadelphia and serves as a reviewer for five peer-reviewed international ophthalmology journals. His scholarly work includes four textbooks and over 90 publications in peer-reviewed journals. Dr. Girach also holds Independent Board Directorships in two biotechnology companies, chairing the R&D committees for both.