gensight biologics

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beursfre
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Re: gensight biologics

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raar koersverloop...
deze morgen naar de 3,055
daarna bergaf
12:48 €2,85
12:49 €2,94 freez en plots....
12:52 €2,78
Inventiva 5,14€ - Pharming 0,95€ - Celyad 6,00€ - Takeaway 80,15€ - Prosus 87,25€
Nanobiotix 14,30€ - Genfit 4,10€ - Innate Pharma 3,05€ - Oxurion 2,35€

Erytech 7,36€
Adocia 10,92€
Nicox 4,62€
Solocal 2,25€



beursfre
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Re: gensight biologics

Bericht door beursfre »

Inventiva 5,14€ - Pharming 0,95€ - Celyad 6,00€ - Takeaway 80,15€ - Prosus 87,25€
Nanobiotix 14,30€ - Genfit 4,10€ - Innate Pharma 3,05€ - Oxurion 2,35€

Erytech 7,36€
Adocia 10,92€
Nicox 4,62€
Solocal 2,25€

beursfre
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Re: gensight biologics

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https://www.gensight-biologics.com/2020 ... reloaded=1

Paris, France, September 28, 2020, 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 announced the recent publication of a new meta-analysis of the natural history of Leber Hereditary Optic Neuropathy (LHON) in the September 2020 issue of the Journal of Neuro-Ophthalmology, the official journal of the North American Neuro-Ophthalmology Society (NANOS).

The paper*, written by leading global authorities on LHON, confirms the low rate of spontaneous visual recovery in patients with a mutated ND4 gene, the most common cause of the disease. For those 15 years or older at onset of visual loss, only an estimated 11.3% experienced some degree of visual recovery.

LHON is a rare, inherited bilaterally blinding disease predominantly affecting otherwise healthy young individuals, mostly men. Some 800 to 1,200 new patients are affected each year in the US and in the EU. Clinicians who manage LHON patients have long agreed that patients only rarely recover their vision once disease onset begins, but they have found it challenging to estimate the rate of spontaneous recovery, given the small number of patients they personally encounter even after decades of clinical practice. The challenge was made more acute because different mutations had different prognoses. Those with the most common mutation, that in the ND4 gene, were generally regarded as having the poorest prognosis.

The Journal of Neuro-Ophthalmology paper, entitled “Visual Outcomes in Leber Hereditary Optic Neuropathy Patients with the m.11778G>A (MTND4) Mitochondrial DNA Mutation”, overcame the challenge of small patient numbers by conducting a meta-analysis of 12 retrospective and 3 prospective studies, which were identified after an extensive review of the scientific and medical literature. Treatment with idebenone did not exclude patients from the sample. This approach enabled the authors to analyze the evolution of visual function in 695 patients with a mutated ND4 gene.

“Many papers have attempted to estimate the extent of spontaneous recovery in LHON, but it has been tricky to arrive at an aggregate rate for the most common underlying mutation, given the different approaches and samples used in the various studies,” commented Dr. Magali Taiel, MD, Chief Medical Officer of GenSight Biologics and a co-author of the paper. “The paper provides an extensive and exhaustive review of the literature and key insights in our knowledge of the disease.”

The estimate of the rate of spontaneous recovery in the paper provides important context for the bilateral improvement observed in the pivotal trials RESCUE and REVERSE for GenSight Biologics’ lead product, LUMEVOQ®, a gene therapy for patients with LHON caused by a mutated ND4 gene. In the trials, the rate of clinically meaningful improvement from nadir of at least 0.3LogMAR, or at least 3 lines on the Snellen chart, was 76% in REVERSE and 71% in RESCUE.

“By reinforcing the insight that spontaneous recovery among LHON patients is rare, this new meta-analysis provides yet more support for bringing LUMEVOQ® to the market,” said Bernard Gilly, Chief Executive Officer of GenSight Biologics. “We are eager to continue working with the authorities to provide a novel treatment to LHON patients.”

On the basis of the efficacy and safety demonstrated by its pivotal trials, other clinical and non-clinical data and an indirect comparison to natural history, LUMEVOQ®’s application for European marketing authorization was submitted in September 2020.

*About the article: Visual Outcomes in Leber Hereditary Optic Neuropathy Patients with the m.11778G>A (MTND4) Mitochondrial DNA Mutation

Running Title: Visual Function in m.11778G>A LHON Patients

Authors: Nancy J Newman, MD1, Valerio Carelli, MD, PhD2,3, Magali Taiel, MD4, Patrick Yu-Wai-Man, MD, PhD5,6,7,8

Affiliations:

1 Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, Georgia USA.

2 IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Bologna, Italy

3 Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy

4 GenSight Biologics, Paris, France.

5Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.

6Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge, UK.

7Moorfields Eye Hospital, London, UK.

8UCL Institute of Ophthalmology, University College London, London, UK.

Dr. Newman, Dr. Carelli and Dr. Yu-Wai-Man are also primary investigators in RESCUE and REVERSE.

Abstract available online at:

https://journals.lww.com/jneuro-ophthal ... 98886.aspx
Inventiva 5,14€ - Pharming 0,95€ - Celyad 6,00€ - Takeaway 80,15€ - Prosus 87,25€
Nanobiotix 14,30€ - Genfit 4,10€ - Innate Pharma 3,05€ - Oxurion 2,35€

Erytech 7,36€
Adocia 10,92€
Nicox 4,62€
Solocal 2,25€

lode78
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Re: gensight biologics

Bericht door lode78 »

goed dat ik nog een klein deel gehouden had moesten ze ontploffen.....
op franse beurssite een koersdoel van 10,5 gezien,

hierdoor twijfel winst te nemen
iemand die ook houder is verdere info?

BioMark
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Re: gensight biologics

Bericht door BioMark »

Even een korte update over Gensight gezien er niet veel gepost wordt over dit aandeel

GenSight Biologics Reports Cash Position as of March 31, 2021 and Provides Operational Update
https://www.gensight-biologics.com/2021 ... al-update/

Publicatie ivm Leber Hereditary Optic Neuropathy (LHON) => GS010
https://www.gensight-biologics.com/2021 ... urnal-eye/

BioMark
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Re: gensight biologics

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GenSight Biologics Announces Nature Medicine Case Report Showing Visual Recovery after GS030 Optogenetic Treatment

First peer-reviewed publication to document partial visual recovery in a blind patient with late-stage inherited retinal disease
GS030 treatment combining gene therapy with light-stimulating medical device enabled patient with 40-year history of retinitis pigmentosa to regain ability to perceive, locate, count and touch objects
Electroencephalographic (EEG) readings during visual tests suggest task-related activity in the visual cortex
Video showing patient successfully performing visual tests available on www.gensight-biologics.com
Paris, France, May 25, 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 announced that the highly-regarded journal Nature Medicine has published the first case report of partial recovery of visual function in a blind patient with late stage retinitis pigmentosa (RP). The subject is a participant in the ongoing PIONEER Phase I/II clinical trial of GenSight Biologics’ GS030 optogenetic therapy. Published in the May issue under the title “Partial recovery of visual function in a blind patient after optogenetic therapy”, the paper* is the first peer-reviewed documentation of visual recovery after a blind patient was treated with optogenetic therapy.

“These are truly groundbreaking findings that move the promise of optogenetics another step from therapeutic concept to clinical use,” commented Bernard Gilly, Co-Founder and Chief Executive Officer of GenSight. “These could not have occurred without the close collaboration we enjoyed with our partners at the Institut de la Vision, the Institute of Ophthalmology Basel and Streetlab. We are especially grateful to the patients who are participating in our trial, whose experiences and input will help us design the next stage of GS030’s clinical development. We will now accelerate the GS030 program to make it our second product to reach the market after LUMEVOQ.”

Optogenetic therapies combine cellular expression of light-sensitive opsins with light stimulation using a medical device. GS030 uses an optimized viral vector (GS030-DP) to express the light-sensitive opsin ChrimsonR in retinal ganglion cells and proprietary light-stimulating goggles (GS030-MD) to project the right wavelength and intensity of light onto the treated retina. GS030-DP is administered via an intravitreal injection.

“It was breathtaking to witness the first recovery of some visual function in a blind patient,” commented Dr. Botond Roska, MD, PhD, last and co-corresponding author and a pioneer in the field of optogenetic vision restoration. Dr. Roska is Founding Director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Co-Founder of GenSight. “We have worked on optogenetic therapy in the lab for 16 years and now seeing the proof of concept in a patient is a unique experience,” he said. “I am most grateful to have shared this long journey with José Sahel, a fellow founder of GenSight; the dedicated team at GenSight; and our other collaborators.”

The subject in the case report, who had been diagnosed with RP 40 years prior to enrollment, had such low visual acuity that prior to receiving GS030, he could only perceive light. His gene therapy injection was followed four and a half months later by training on the use of the GS030-MD device. Seven months after the start of his training, he began to report signs of visual improvement. Visual function tests showed he acquired the ability to perceive, locate, count and touch objects when his treated eye was stimulated with the GS030-MD goggles. Without the goggles, he could not perform the tasks.

While the patient performed vision-oriented tasks, recordings were taken using extracranial multi-channel electroencephalography (EEG), a non-invasive technique that provides a readout of neuronal activity across the cortex. The EEG signals suggest that the act of carrying out the visual perception tests was accompanied by neurophysiological activity in the visual cortex.

In addition, the patient also reported significant improvements in his ability to conduct day-to-day activities such as navigating in outdoor and indoor environments and detecting household objects and furniture.

“Watching a patient benefit for the first time from this trial using optogenetics to treat blindness has been a uniquely rewarding experience,” commented Dr. José-Alain Sahel, MD, PhD, lead and co-corresponding author, Co-Founder of GenSight, and Founder of the Institut de la Vision (Sorbonne-Université/Inserm/CNRS), Paris, France. Dr. Sahel is also Director of Institut Hospitalo-Universitaire FOReSIGHT, Paris, France, and Distinguished Professor and Chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA. He added, “Being able to take part in bringing this new scientific approach to the clinic reflects the long-term collaboration with Botond Roska, the scientists of the Vision Institute, our clinicians, the Streetlab and psychophysics teams, and GenSight.”

A video of the patient performing the tests, which was submitted as supplementary material to Nature Medicine, can be viewed at www.gensight-biologics.com.



Key Opinion Leader Webcast: June 4, 2021 at 2:00 PM CEST/8:00 AM EDT

Dr. Sahel and Dr. Roska will discuss the case report on a KOL webcast dedicated to Optogenetics and GS030 and hosted by GenSight Biologics.

Details will be announced at a later date.

Detailed findings can be found at https://www.nature.com/articles/s41591-021-01351-4.

https://www.gensight-biologics.com/2021 ... treatment/









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