Pioneering Protein Splicing:
A Novel Gene Therapy Modality
SpliceBio Announces U.S. FDA IND Clearance of SB-007 to Commence Phase 1/2 Clinical Study in Patients with Stargardt Disease
BARCELONA, SPAIN, 12 December 2024 – SpliceBio, a genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for lead program SB-007. SB-007 is the only clinical-stage therapeutic addressing the root genetic cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations.
MANAGEMENT TEAM
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.