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GENSIGHT BIOLOGICS REPORTS SECOND PATIENT CASE SHOWING SIGNIFICANT VISUAL
RECOVERY AFTER GS030 OPTOGENETIC TREATMENT


17 November 2021
 * Press releases

 * Vision improved significantly, from being barely able to perceive light
   before treatment to being able to locate and count objects, one year after
   gene therapy
 * Video showing patient successfully performing visual tests available on
   www.gensight-biologics.com

Paris, France, November 17, 2021, 7:30 am CEST – 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 reported
a second case of a patient with late-stage retinitis pigmentosa (RP) who
partially recovered her visual function after treatment with GS030 optogenetic
therapy.

 

“We are delighted by the highly encouraging signs emerging from PIONEER, which
are demonstrating the promise of optogenetics to treat one of the most common
blinding genetic disorders,” commented Bernard Gilly, Co-Founder and Chief
Executive Officer of GenSight. “We will eagerly push forward towards the
realization of a treatment for retinitis pigmentosa patients.”

 

The GS030 optogenetic treatment, which combines gene therapy with the use of
light-stimulating goggles, led to the patient being able to perceive and count
objects one year after injection with GS030’s gene therapy component. The
patient is a participant in the ongoing PIONEER Phase I/II clinical trial of
GS030, like another patient whose partial recovery was published as a case
report in Nature Medicine in May 2021.

 

The patient, whose improvement is documented in a video, had been diagnosed with
retinitis pigmentosa 20 years prior to enrollment and, at the time of injection,
was barely able to perceive light. She received a single intravitreal (IVT)
injection with the medium dose (1.5E11 vector genomes) of GS030 gene therapy in
her worse-seeing eye and, after four months, started training on the use of the
device.

 

Twelve months after injection, the patient could detect and correctly locate
objects of different sizes and contrasts placed on a white table in front of
her, with a 57% success rate when wearing the GenSight light-stimulating
goggles, compared to only 24% without the device.

 

A video of the patient performing the tests can be viewed at
www.gensight-biologics.com.

 

The patient was featured in an update on PIONEER that was presented by Dr.
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, at the Retina Sub-Specialty
Day of the American Academy of Ophthalmology (AAO) annual meeting (November
12-15, 2021).

 

Dr. Sahel also provided an update on the safety of GS030. The optogenetic
therapy has been well-tolerated up to 3 years after the single intravitreal
injection administered to the 9 subjects treated to date. There have been no
systemic issues related to gene therapy, no adverse events leading to study
discontinuation, and no withdrawal of participants.

 

In September 2021, after reviewing the safety data for the first three cohorts
of the PIONEER trial, the independent Data Safety Monitoring Board recommended
the use of the highest dose of the gene therapy (5E11 vector genomes) in the
extension cohort. The trial’s topline results are expected to be available in H2
2022.

 

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.

About GS030

GS030 leverages GenSight Biololgics’ optogenetics technology platform, a novel
approach to restore vision in blind patients using a combination of ocular gene
therapy and tailored light-activation of treated retinal cells. The gene
therapy, which is delivered via a single intravitreal injection, introduces a
gene encoding for a light-sensitive protein (ChrimsonR-tdT) into retinal
ganglion cells, making them responsive to light and bypassing photoreceptors
killed off by diseases such as retinitis pigmentosa (RP). Because ChrimsonR-tdT
is activated by high intensities of amber light, a wearable medical device is
needed to stimulate the treated retina. The optronic light‑stimulating goggles
(GS030-MD) encode the visual scene in real-time and project a light beam with a
specific wavelength and intensity onto the treated retina. Treatment with GS030
requires patients to wear the external wearable device in order to enable
restoration of their visual function. With the support of the Institut de la
Vision in Paris and the team of Dr. Botond Roska at the Friedrich Miescher
Institute in Basel, GenSight is investigating GS030 as therapy to restore vision
in patients suffering from late-stage RP. GenSight’s optogenetics approach is
independent of the specific genetic mutations causing blindness and has
potential applications in other diseases of the retina in which photoreceptors
degenerate, like dry age‑related macular degeneration (dry-AMD). GS030 has been
granted Orphan Drug Designation in the United States and Europe.

About Optogenetics

Optogenetics is a biological technique that involves the transfer of a gene
encoding for a light sensitive protein to cause neuronal cells to respond to
light stimulation. As a neuromodulation method, it can be used to modify or
control the activities of individual neurons in living tissue and even in-vivo,
with a very high spatial and temporal resolution. Optogenetics combines (1) the
use of gene therapy methods to transfer a gene into target neurons with (2) the
use of optics and electronics (optronics) to deliver the light to the transduced
cells. Optogenetics holds clinical promise in the field of vision impairment or
degenerative neurological disorders.

About Retinitis Pigmentosa

Retinitis pigmentosa (RP) is a family of orphan genetic diseases caused by
multiple mutations in numerous genes involved in the visual cycle. Over 100
genetic defects have been implicated. RP patients generally begin experiencing
vision loss in their young adult years, with progression to blindness by age 40.
RP is the most widespread hereditary cause of blindness in developed nations,
with a prevalence of about 1.5 million people throughout the world. In Europe
and the United States, about 350,000 to 400,000 patients suffer from RP, and
every year between 15,000 and 20,000 new patients with RP lose sight. There is
currently no curative treatment for RP.

About the PIONEER Phase I/II trial

PIONEER is a first-in-man, multi-center, open label dose-escalation study to
evaluate the safety and tolerability of GS030 in 12-18 subjects with late-stage
retinitis pigmentosa. GS030 combines a gene therapy (GS030-DP) administered via
a single intravitreal injection with a wearable optronic visual stimulation
device (GS030-MD). Eligible patients in the first three cohorts are those
affected by end-stage non-syndromic RP with no light perception (NLP) or light
perception (LP) levels of visual acuity. The extension cohort will include
patients with hand motion (HM) and counting fingers (CF) levels of visual
acuity.

 

As per protocol, three cohorts with three subjects each will be administered an
increasing dose of GS030-DP via a single intravitreal injection in their
worse-seeing eye. An extension cohort will receive the highest tolerated dose.
The DSMB will review the safety data of all treated subjects in each cohort and
will make recommendations before a new cohort receives the next dose. The
primary outcome analyses will be on the safety and tolerability at one year
post-injection. PIONEER is being conducted in three centers in the United
Kingdom, France and the United States.

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CONTACTS

GenSight Biologics
Chief Financial Officer
Thomas Gidoin
+33 (0)1 76 21 72 20
tgidoin@gensight-biologics.com
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Media Relations
Jeanene Timberlake
+1 646-770-8858
jtimberlake@rooneyco.com
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Investor Relations
Guillaume van Renterghem
+33 (0)6 69 99 37 83
gvanrenterghem@lifesciadvisors.com
Orpheon Finance
Retail Investors
James Palmer
+33 7 60 92 77 74
j.palmer@orpheonfinance.com


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