Business Wire

GenSight Biologics Announces Additional Promising Results of Its Phase I/II Study with GS010 for the Treatment of Leber’s Hereditary Optic Neuropathy (LHON)


GenSight Biologics, a biotechnology company discovering and developing novel gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, announces additional promising results of its Phase I/II study, designed to demonstrate the safety and tolerability of GS010 in 15 patients with Leber’s Hereditary Optic Neuropathy (LHON).

This Smart News Release features multimedia. View the full release here:

Note (*): Excludes "hand motion" patients (hand movements between 30cm and 1m), in accordance with t ...

Note (*): Excludes "hand motion" patients (hand movements between 30cm and 1m), in accordance with the Phase III protocol. Source: Company (Graphic: Business Wire)

Each cohort of three patients was administered an escalating dose of GS010 through a single intravitreal injection in the eye most severely affected by the disease. Recruitment was completed in April 2015. These patients had an average onset of disease of 6 years.

At 48 weeks post-injection, in patients with an onset of disease of less than 2 years, a gain of +30 letters

(-0.59 LogMAR) was observed in the treated eye and +13 letters (-0.25 LogMAR) in the untreated eye, a difference of 17 letters in favor of the treated eye. No significant difference was observed in patients with an onset of disease of more than two years.

Bernard Gilly, CEO and co-founder of GenSight, commented, "This preliminary data is very encouraging and validates the design of the two Phase III studies currently ongoing in the US and Europe on GS010 for the treatment of Leber’s Hereditary Optic Neuropathy, a neurodegenerative retinal disease. Moreover, these results support the potential of our mitochondrial targeting sequence technology platform,or MTS, and allows us to envision other applications in diseases involving defects of the mitochondrion, in ophthalmology as well as in other therapeutic areas."

GS010 targets LHON, a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. GS010 leverages a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research works conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produce the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.

Dr. Catherine Vignal, investigator of the study and head chief at the Rothschild Foundation Hospital in Paris, added, "These preliminary data at 48 weeks give significant hope to patients suffering from LHON, a very severe disease with no existing curative treatment.”

GenSight Biologics is currently conducting two Phase III clinical studies (Rescue and Reverse) in Europe and the United States to assess the efficacy of GS010 in patients affected with LHON due to the ND4 mutation, with an onset of vision loss of less than one year. The top-line results at 48 weeks follow-up are expected in late 2017.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible vision loss in teens and young adults. 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 in order to offer patients a sustainable functional visual recovery.

Contact information

GenSight Biologics
Thomas Gidoin, +33 (0) 6 01 36 35 43
Chief Financial Officer
Media Relations
Marion Janic, +1-212-223-4017

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

JustPremium Launches Rich Video Product Suite19.3.2018 08:00Pressemelding

Programmatic rich media and video ad marketplace, JustPremium, has today announced the launch of a new product suite, Rich Video. The new offering allows clients to build multiple rich video ad units with a single click using the advertisers existing assets. Using Rich Video allows advertisers to create an engaging advert, leading to higher engagement and interaction rates. Ultimately these formats help create a better user experience and a higher return on investment. JustPremium launches this product at a time when quality video inventory has never been more in demand, and brand-safe video inventory that advertisers require has never been in shorter supply. Rich Video allows publishers to build multiple creative ad units in minutes, enabling them to test different options and optimise their message to give the best performance. By utilising the assets that have already been created for Facebook, Adwords and YouTube, this process allows advertisers to supercharge their existing conten

GovSat-1 Satellite Goes Operational19.3.2018 07:40Pressemelding

GovSat-1 enters operational service today, to provide secure communications to governmental and institutional users. GovSat-1 is the first satellite of GovSat, a public private partnership between the Government of Luxembourg and the world-leading satellite operator SES. The satellite was launched into space on 31 January on board a flight-proven SpaceX Falcon 9 rocket from Cape Canaveral Air Force Station, and has since undergone extensive testing. This press release features multimedia. View the full release here: GovSat-1 launch. (Photo: SpaceX) The multi-mission satellite is operated by GovSat from the Secure Mission Operations Centre in Luxembourg. The highly flexible and resilient GovSat-1 payload uses dedicated frequencies in X-band and military Ka-band. It enables an array of applications such as connectivity for theatres of operation, interconnection of institutional or defence sites, border control, Intelligence, Surve

Digi Communications N.V.: Announcement on the Convocation Date for the Company’s GSM Approving the 2017 Annual Report19.3.2018 07:34Pressemelding

The Company would like to inform its investors and the market that the Board of Directors anticipates that on Wednesday, 21 March 2018, it will convene the general shareholders meeting (the “GSM”) called to approve, among others, the 2017 Annual Report. On the same date, the Company will make public, among others, its 2017 Annual Report and the auditor’s report. In accordance with the provisions of the articles of association of the Company, the GSM is scheduled to take place on 2 May 2018. Therefore, we would like to inform you with respect to the update to the Company’s Financial Reporting Calendar for 2018, which is available on Digi’s website. For details, please access the Digi’s website: (Investor Relations Section). About Digi Communications NV Digi is the parent holding company of RCS & RDS, a leading provider of pay TV and telecommunications services in Romania and Hungary. In addition, RCS & RDS provides mobile services as an MVNO to the large Roman

Dai Nippon Printing Chooses Gemalto’s Biometric Facial Recognition Solution to Facilitate Mobile Banking Access in Japan19.3.2018 06:00Pressemelding

Gemalto, the world leader in digital security, today announced that it has been selected by Dai Nippon Printing (DNP), a financial solutions provider in Japan, to make mobile banking transactions easier to use. Leveraging Gemalto’s facial biometric authentication solution - Mobile Protector- DNP has started to offer facial recognition to secure access to its mobile banking apps. This enables banking customers to easily and quickly log on to mobile banking services upon successful facial authentication, providing a more convenient user experience. This press release features multimedia. View the full release here: Facial recognition for mobile banking authentication. (Photo: Gemalto) Gemalto’s 2017 eBanking Trends research found that 68 percent of banks plan to implement facial recognition over the next five years. 80 percent of surveyed consumers perceived biometrics to be more secure than the traditional username and password co

Gemalto Helps European Banks Build Business Opportunities from PSD219.3.2018 06:00Pressemelding

Gemalto, the world leader in digital security, believes that the implementation of PSD2 (Payment Services Directive 2) offers European banks a unique opportunity to deliver digital innovation, robust protection and seamless user experience that will be critical in a new era of open markets. The Regulatory Technical Standards (RTS) have just been published in the EU Official Journal, and detail the responsibilities and obligations of all banking and payment stakeholders concerning access to account, customer data protection, user convenience, and payment security. Gemalto believes that long term success will be built around solutions tailored to individual requirements, whilst drawing on products and services already proven to deliver the core principles of PSD2. This press release features multimedia. View the full release here: Solutions to help financial institutions for implementation of PSD2. Credit: istockphoto Adoption of t

ExThera Medical Announces Creation of European Subsidiary19.3.2018 05:23Pressemelding

ExThera Medical Corporation, a developer of therapies for removing bacteria and viruses from blood, today announced the company’s plan to create ExThera Medical Europe and appointed Carla Kikken-Jussen as managing director of the new wholly-owned subsidiary. Carla brings more than 30 years of experience in clinical research and the commercialization of new medical devices in the European market to ExThera Medical, where she will oversee the strategic direction for ExThera’s European operations. “The establishment of our European subsidiary is an exciting milestone as we prepare for commercialization,” said Robert Ward, President and CEO of ExThera Medical. “Carla has a distinguished track record in medical device development and commercialization, including overseeing clinical research, regulatory affairs, quality assurance, and operations. As CEO of her company, Meditech, she successfully supported the rapid clinical adoption of many new medical devices. Carla’s strategic leadership w