Environmental & Social Information • Feb 28, 2023
Environmental & Social Information
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Press Release
Paris, France, February 28, 2023, 7:30 am CET – 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, highlights its commitment to fight rare neurodegenerative diseases by raising awareness of Leber Hereditary Optic Neuropathy (LHON) at Rare Disease Day through the launch of a European awareness campaign in Q1 2023, in close collaboration with LHON experts.
Rare Disease Day is a globally coordinated movement aiming to raise awareness for the 300 million people living with a rare disease worldwide. GenSight Biologics is pleased to join the movement and take this opportunity to raise awareness for LHON, a rare disease with significant unmet need.
LHON is a relatively unknown disease that strikes people in the prime of life. It is characterized by rapid, usually irreversible, painless, and severe vision loss1-4 .
This condition is thought to affect from one out of 30,000 to one out of 50,000 people in Europe 1-3 . Though women are also affected by the condition, LHON most often impacts young men2 . Onset typically occurs between ages 15 and 35, but the disease has been observed in patients from 2 to 87 years of age 1 .
Patients typically lose central vision in one eye and within a few weeks or months, vision begins to degrade in the second eye 5 . Visual acuity eventually stabilizes at a very low visual acuity 1 , the natural progression of the disease leads to legal blindness 6 .
Patients are affected physically but also socially and mentally7,8 . The deterioration of central vision severely impedes the ability to carry out day-to-day activities such as reading, writing and cooking. Social interactions become more difficult, and individual pastimes become more challenging to pursue. For many
patients, the loss of autonomy is an emotional blow. Taking a walk, become nearly impossible 7-9 for example. Patients also report feeling psychological impacts from the disease.
In an online survey, nearly 22% of LHON patients reported thoughts of suicide after experiencing bilateral vision loss 10 .
"I was told that I had LHON, that it was a genetic disease transmitted by the mother, that it was rare and that I would lose my sight in three months. Everything is slipping away: I won't be able to drive, I won't be able to see my wife's face, I won't be able to distinguish colors or what I eat". LHON patient
Though often forgotten, caregivers may also find themselves affected by LHON as well9 . They might find their lives upended as a result of the demands of the disease, and caregivers report feeling compelled to put careers on hold or to reorganize their lives in order to accompany the patient day in and day out 9 .
LHON's impact is felt beyond the immediate social circle of the patient. Other costs associated with the condition include increased medical costs due to repeat hospital visits and health care administration, as well as the cost of low-vision aids, home-based care, and modifications to a patient's home 11 .
Estimates of the annual productivity loss from legal blindness run to \$411 billion8 .
LHON, like many rare diseases, is often overlooked and diagnosis delayed because it can be confused with more well-known conditions like optic neuritis (multiple sclerosis) and retinopathy12,13 . It is a psychological tool for the patients and their loved ones who are often sent to various physicians before to finally obtain a definitive diagnosis thanks to genetic testing. Interventions that support psychological health, reinforce coping mechanisms, and change illness perceptions are critical to patients with LHON 9 . Additionally, intervention to educate patients on visual aid technologies may help improve their psychological well-being, and local patient groups and charities can also provide support to those with visual impairment 14,15 .
As a healthcare company, GenSight has a responsibility that goes far beyond giving access to breakthrough solutions to patients. We are committed to actively support and help to improve the care of patients suffering from rare ophthalmic diseases, such as LHON. The Company has therefore developed, in close collaboration with LHON experts, an awareness campaign to educate and increase the understanding of this devastating disease. The ultimate goal: accelerate time to diagnosis.
The campaign will be launched in the top 4 EU countries and UK in Q1 2023 by the GenSight local teams in the countries and is addressed to ophthalmologists and neurologists.
Any person suspecting to be suffering from LHON should contact his health care professional and seek support from the local patient groups.
Corporate Communications Director Clothilde Caillet ccaillet@gensight-biologics.com
Investor Relations Guillaume van Renterghem [email protected] +41 (0)76 735 01 31
Media Relations Jeanene Timberlake [email protected] +1 646-770-8858
Retail Investors James Palmer [email protected] +33 (0)7 60 92 77 74
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.
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