Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that clinical data of LUMEVOQ® and GS030 gene therapies will be presented at the 125th Annual Meeting of the American Academy of Ophthalmology (AAO) in New Orleans, Louisiana (November 12-15, 2021).

Retina Subspecialty Day @ American Academy of Ophthalmology (AAO)
November 12, 2021
Morial Convention Center, New Orleans

José-Alain Sahel, MD, Co-founder of GenSight Biologics and of the Institut de la Vision (Sorbonne-Université/Inserm/CNRS), Paris, France, and Distinguished Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine, Pittsburgh, PA, USA, will discuss the reported signs of efficacy from a second patient affected by Retinitis Pigmentosa and treated with GS030 in the Phase I/IIa trial PIONEER. A first case report was published in Nature Medicine in May 2021.

“Optogenetics in the Clinic: Safety and Efficacy Updates on the Phase I/II Clinical Trial PIONEER”

Oral Presentation by José-Alain Sahel, MD Session: RET09 - Section VII: Late-Breaking Developments, Part I Venue: The Great Hall Friday, November 12, 2021, 3:11 - 3:16 pm CDT

American Academy of Ophthalmology (AAO)
November 12-15, 2021
Morial Convention Center, New Orleans

Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, GA, USA, and the International Coordinating Investigator of the REFLECT Phase III trial of LUMEVOQ®, will discuss the findings from the REFLECT trial in the context of Leber Hereditary Optic Neuropathy (LHON) natural history and the two other clinical trials RESCUE and REVERSE.

“The Phase 3 REFLECT Trial: Efficacy and Safety of Bilateral Gene Therapy for LHON”

Oral Presentation by Nancy Newman, MD Abstract number: PA050 Venue: Room 255-257 Monday, November 15, 2021, 8:48 - 8:55 am CDT

Sean P. Donahue, MD, PhD, Coleman Professor and Vice Chair for Clinical Affairs, and Chief, Pediatric Ophthalmology Department at the Vanderbilt Children’s Hospital in Nashville, TN, USA, and an International Principal Investigator in the REFLECT Phase III trial of LUMEVOQ®, will discuss the findings from 6 patients affected by ND4 LHON and bilaterally treated with LUMEVOQ® in an FDA-approved compassionate use protocol.

“Initial Results From Bilateral Gene Therapy for LHON 11778 Mutation in a Compassionate Use Protocol”

Poster Presentation by Sean Donahue, MD, PhD Abstract number: PO014 Saturday, November 13, 2021, 12:45 - 12:50 pm CDT

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, LUMEVOQ® (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

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