Latest news with #ANSM
Yahoo
12-06-2025
- Business
- Yahoo
GenSight Biologics Reaches Agreement with ANSM for Opening of LUMEVOQ® Named Early Access Program (AAC) in France
Agreement to consider opening the French Early Access Program expeditiously after approval of a dose-ranging study Design for dose-ranging study submitted, with clinical trial application targeted for Q3 2025 Opening of AAC targeted for Q4 2025 at the latest Multiple near-term value catalysts expected over next 12 months PARIS, June 12, 2025--(BUSINESS WIRE)--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (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 it has reached agreement with the French medicines safety agency ANSM to consider expeditiously opening the French named early access program (AAC) for LUMEVOQ® upon approval of a dose-ranging clinical study. Clear Path Forward for LUMEVOQ® AAC Program in France Following extensive discussions, GenSight reached alignment with ANSM on a plan to open the LUMEVOQ® AAC program. The agency has agreed that the authorization of a focused dose-ranging study could enable the opening of the early access program. The Company has submitted a preliminary design of the study to the agency and aims to finalize the protocol of a dose-ranging study in Q3 2025. In parallel, the Company will work with the agency to find a solution for patients who may not be included in the study but who may benefit from the AAC program. The AAC program is targeted to open in Q4 2025 at the latest. "In the interest of clearing the way for patients to be treated with LUMEVOQ, we are pleased to have achieved this important agreement with ANSM on a clear path forward for opening patient access to LUMEVOQ in France" said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. "We are thankful for the ANSM's ongoing engagement and openness to discussion, as we work towards the shared goal of providing access to LHON patients who urgently need a safe and effective treatment." Financing Strategy to Support Regulatory Milestones GenSight is implementing a financing strategy to bridge operations through the opening of the AAC program and prepare for the new global Phase III clinical trial. The Company is actively pursuing various funding initiatives to support these critical regulatory milestones. "We are working on our financing strategy to manage the bridge between now and the opening of the AAC program and the initiation of the Phase III study," commented Jan Eryk Umiastowski, CFO of GenSight Biologics. "Our focus remains on securing the resources needed to execute our regulatory strategy while exploring additional funding opportunities. " Advancing Core Marketing Authorization Strategy In parallel with the French AAC program developments, GenSight will continue to advance its global marketing authorization strategy: Preparation for regulatory consultations in the US and EU Planning for the global Phase III trial scheduled to begin in 2026, designed to meet FDA and EMA requirements Completing the transition to a new manufacturing partner to secure reliable clinical and commercial supply Advancing preparations for MHRA submission in the United Kingdom During this period, the Company will actively pursue additional non-dilutive funding opportunities, including licensing arrangements outside North America and Europe and potential M&A activities. Multiple Value Catalysts GenSight anticipates a number of significant value-creating events in 2025-2026: Q3 2025: Clinical trial application for focused dose-ranging study Q4 2025: Completion of manufacturing tech transfer Q4 2025: Opening of the AAC program Q2 2026: Completion of Phase III trial preparations Early H2 2026: Initiation of global Phase III clinical trial Early H2 2026: pre-submission meeting with MHRA Financial Position The Company received €0.7 million of its Research Tax Credit (CIR) end of May, with the remaining €0.4 million to be paid in July 2025. Based on current operations and projections, GenSight's cash will support operations until mid-July 2025. The Company acknowledges that current funds are insufficient to cover operational requirements for the next 12 months. The planned financing initiatives are designed to extend the cash runway and enable the initiation of the Phase III clinical trial and UK MHRA marketing application for LUMEVOQ®. As of June 12, 2025, the number of outstanding GenSight Biologics shares was 131,466,495 ordinary shares. Risk factors Detailed information regarding the Company, including its business, financial information, results, perspectives and related risk factors are contained (i) in the Company's 2024 Universal Registration Document filed with the AMF on April 8, 2025 under number D.25-0234. This document, as well as other regulated information and all of the Company's press releases, can be accessed on the Company's website ( and/or AMF ( The reader's attention is drawn to the risk factors related to the Company and its activities presented in chapter 3 of its 2024 URD in particular the liquidity risk presented in the chapter 3.1.1. 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, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness 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 to offer patients a sustainable functional visual recovery. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding product development prospects and financial projections. These statements do not constitute guarantees of future performance and involve risks and uncertainties. While the Company is actively pursuing financing solutions, regulatory requirements necessitate disclosure that material uncertainty exists regarding the Company's ability to continue as a going concern if sufficient funding cannot be secured. A further list and description of risks and uncertainties that could cause actual results to differ materially from those set forth in the forward-looking statements in this press release can be found in GenSight Biologics' regulatory filings with the French Autorité des Marchés Financiers. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, GenSight Biologics undertakes no obligation to update or revise the information contained in this press release. View source version on Contacts GenSight Biologics Chief Financial Officer Jan Eryk Umiastowski jeumiastowski@


Business Wire
12-06-2025
- Business
- Business Wire
GenSight Biologics Reaches Agreement with ANSM for Opening of LUMEVOQ ® Named Early Access Program (AAC) in France
PARIS--(BUSINESS WIRE)--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (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 it has reached agreement with the French medicines safety agency ANSM to consider expeditiously opening the French named early access program (AAC) for LUMEVOQ ® upon approval of a dose-ranging clinical study. Clear Path Forward for LUMEVOQ ® AAC Program in France Following extensive discussions, GenSight reached alignment with ANSM on a plan to open the LUMEVOQ ® AAC program. The agency has agreed that the authorization of a focused dose-ranging study could enable the opening of the early access program. The Company has submitted a preliminary design of the study to the agency and aims to finalize the protocol of a dose-ranging study in Q3 2025. In parallel, the Company will work with the agency to find a solution for patients who may not be included in the study but who may benefit from the AAC program. The AAC program is targeted to open in Q4 2025 at the latest. "In the interest of clearing the way for patients to be treated with LUMEVOQ, we are pleased to have achieved this important agreement with ANSM on a clear path forward for opening patient access to LUMEVOQ in France" said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics."We are thankful for the ANSM's ongoing engagement and openness to discussion, as we work towards the shared goal of providing access to LHON patients who urgently need a safe and effective treatment." Financing Strategy to Support Regulatory Milestones GenSight is implementing a financing strategy to bridge operations through the opening of the AAC program and prepare for the new global Phase III clinical trial. The Company is actively pursuing various funding initiatives to support these critical regulatory milestones. ' We are working on our financing strategy to manage the bridge between now and the opening of the AAC program and the initiation of the Phase III study,' commented Jan Eryk Umiastowski, CFO of GenSight Biologics. "Our focus remains on securing the resources needed to execute our regulatory strategy while exploring additional funding opportunities." Advancing Core Marketing Authorization Strategy In parallel with the French AAC program developments, GenSight will continue to advance its global marketing authorization strategy: Preparation for regulatory consultations in the US and EU Planning for the global Phase III trial scheduled to begin in 2026, designed to meet FDA and EMA requirements Completing the transition to a new manufacturing partner to secure reliable clinical and commercial supply Advancing preparations for MHRA submission in the United Kingdom During this period, the Company will actively pursue additional non-dilutive funding opportunities, including licensing arrangements outside North America and Europe and potential M&A activities. Multiple Value Catalysts GenSight anticipates a number of significant value-creating events in 2025-2026: Financial Position The Company received €0.7 million of its Research Tax Credit (CIR) end of May, with the remaining €0.4 million to be paid in July 2025. Based on current operations and projections, GenSight's cash will support operations until mid-July 2025. The Company acknowledges that current funds are insufficient to cover operational requirements for the next 12 months. The planned financing initiatives are designed to extend the cash runway and enable the initiation of the Phase III clinical trial and UK MHRA marketing application for LUMEVOQ ®. As of June 12, 2025, the number of outstanding GenSight Biologics shares was 131,466,495 ordinary shares. Risk factors Detailed information regarding the Company, including its business, financial information, results, perspectives and related risk factors are contained (i) in the Company's 2024 Universal Registration Document filed with the AMF on April 8, 2025 under number D.25-0234. This document, as well as other regulated information and all of the Company's press releases, can be accessed on the Company's website ( and/or AMF ( The reader's attention is drawn to the risk factors related to the Company and its activities presented in chapter 3 of its 2024 URD in particular the liquidity risk presented in the chapter 3.1.1. 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, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness 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 to offer patients a sustainable functional visual recovery. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding product development prospects and financial projections. These statements do not constitute guarantees of future performance and involve risks and uncertainties. While the Company is actively pursuing financing solutions, regulatory requirements necessitate disclosure that material uncertainty exists regarding the Company's ability to continue as a going concern if sufficient funding cannot be secured. A further list and description of risks and uncertainties that could cause actual results to differ materially from those set forth in the forward-looking statements in this press release can be found in GenSight Biologics' regulatory filings with the French Autorité des Marchés Financiers. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, GenSight Biologics undertakes no obligation to update or revise the information contained in this press release.
Yahoo
12-06-2025
- Business
- Yahoo
GenSight Biologics Reaches Agreement with ANSM for Opening of LUMEVOQ® Named Early Access Program (AAC) in France
Agreement to consider opening the French Early Access Program expeditiously after approval of a dose-ranging study Design for dose-ranging study submitted, with clinical trial application targeted for Q3 2025 Opening of AAC targeted for Q4 2025 at the latest Multiple near-term value catalysts expected over next 12 months PARIS, June 12, 2025--(BUSINESS WIRE)--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (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 it has reached agreement with the French medicines safety agency ANSM to consider expeditiously opening the French named early access program (AAC) for LUMEVOQ® upon approval of a dose-ranging clinical study. Clear Path Forward for LUMEVOQ® AAC Program in France Following extensive discussions, GenSight reached alignment with ANSM on a plan to open the LUMEVOQ® AAC program. The agency has agreed that the authorization of a focused dose-ranging study could enable the opening of the early access program. The Company has submitted a preliminary design of the study to the agency and aims to finalize the protocol of a dose-ranging study in Q3 2025. In parallel, the Company will work with the agency to find a solution for patients who may not be included in the study but who may benefit from the AAC program. The AAC program is targeted to open in Q4 2025 at the latest. "In the interest of clearing the way for patients to be treated with LUMEVOQ, we are pleased to have achieved this important agreement with ANSM on a clear path forward for opening patient access to LUMEVOQ in France" said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. "We are thankful for the ANSM's ongoing engagement and openness to discussion, as we work towards the shared goal of providing access to LHON patients who urgently need a safe and effective treatment." Financing Strategy to Support Regulatory Milestones GenSight is implementing a financing strategy to bridge operations through the opening of the AAC program and prepare for the new global Phase III clinical trial. The Company is actively pursuing various funding initiatives to support these critical regulatory milestones. "We are working on our financing strategy to manage the bridge between now and the opening of the AAC program and the initiation of the Phase III study," commented Jan Eryk Umiastowski, CFO of GenSight Biologics. "Our focus remains on securing the resources needed to execute our regulatory strategy while exploring additional funding opportunities. " Advancing Core Marketing Authorization Strategy In parallel with the French AAC program developments, GenSight will continue to advance its global marketing authorization strategy: Preparation for regulatory consultations in the US and EU Planning for the global Phase III trial scheduled to begin in 2026, designed to meet FDA and EMA requirements Completing the transition to a new manufacturing partner to secure reliable clinical and commercial supply Advancing preparations for MHRA submission in the United Kingdom During this period, the Company will actively pursue additional non-dilutive funding opportunities, including licensing arrangements outside North America and Europe and potential M&A activities. Multiple Value Catalysts GenSight anticipates a number of significant value-creating events in 2025-2026: Q3 2025: Clinical trial application for focused dose-ranging study Q4 2025: Completion of manufacturing tech transfer Q4 2025: Opening of the AAC program Q2 2026: Completion of Phase III trial preparations Early H2 2026: Initiation of global Phase III clinical trial Early H2 2026: pre-submission meeting with MHRA Financial Position The Company received €0.7 million of its Research Tax Credit (CIR) end of May, with the remaining €0.4 million to be paid in July 2025. Based on current operations and projections, GenSight's cash will support operations until mid-July 2025. The Company acknowledges that current funds are insufficient to cover operational requirements for the next 12 months. The planned financing initiatives are designed to extend the cash runway and enable the initiation of the Phase III clinical trial and UK MHRA marketing application for LUMEVOQ®. As of June 12, 2025, the number of outstanding GenSight Biologics shares was 131,466,495 ordinary shares. Risk factors Detailed information regarding the Company, including its business, financial information, results, perspectives and related risk factors are contained (i) in the Company's 2024 Universal Registration Document filed with the AMF on April 8, 2025 under number D.25-0234. This document, as well as other regulated information and all of the Company's press releases, can be accessed on the Company's website ( and/or AMF ( The reader's attention is drawn to the risk factors related to the Company and its activities presented in chapter 3 of its 2024 URD in particular the liquidity risk presented in the chapter 3.1.1. 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, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness 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 to offer patients a sustainable functional visual recovery. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding product development prospects and financial projections. These statements do not constitute guarantees of future performance and involve risks and uncertainties. While the Company is actively pursuing financing solutions, regulatory requirements necessitate disclosure that material uncertainty exists regarding the Company's ability to continue as a going concern if sufficient funding cannot be secured. A further list and description of risks and uncertainties that could cause actual results to differ materially from those set forth in the forward-looking statements in this press release can be found in GenSight Biologics' regulatory filings with the French Autorité des Marchés Financiers. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, GenSight Biologics undertakes no obligation to update or revise the information contained in this press release. View source version on Contacts GenSight Biologics Chief Financial Officer Jan Eryk Umiastowski jeumiastowski@


Time of India
05-06-2025
- Business
- Time of India
Gland Pharma shares in focus after subsidiary Cenexi gets 11 observations from French regulator
Gland Pharma shares will be in focus on Thursday after the company announced that its material subsidiary, Cenexi, received 11 observations from France's drug regulator, Agence nationale de sécurité du médicament et des produits de santé (ANSM), following a Good Manufacturing Practices (GMP) inspection at its Fontenay manufacturing facility. In a stock exchange filing, Gland Pharma stated: 'The ANSM, France, has issued its final report citing 11 observations pursuant to the GMP inspection of Cenexi's Fontenay manufacturing facility conducted between December 9 and December 19, 2024.' In response, Cenexi has submitted a Corrective and Preventive Action (CAPA) plan, outlining measures to be implemented over a 3- to 12-month period, depending on the nature of each observation. The plan has been accepted by ANSM. Also Read: MRF snatches India's highest-priced stock crown back from Elcid Investments Live Events The company clarified that the observations will not impact manufacturing operations at the Fontenay site. Gland Pharma added that Cenexi is committed to resolving the issues in coordination with the French regulator and will continue to update stock exchanges on further developments. Also Read: These 10 Nifty microcap stocks can rally 70-200% in the next 12 months Gland Pharma Share Price Target and Performance According to Trendlyne, the average target price for Gland Pharma is Rs 1,678, implying a potential upside of 3% from current levels. Of the 12 analysts tracking the stock, the consensus rating is 'Hold'. On Wednesday, Gland Pharma shares closed 1.88% higher at Rs 1,623.7 on the BSE, while the Sensex gained 0.32%. The stock has rallied 16% in the last one month but remains down 43% over the past three years. The company's market capitalisation stands at Rs 26,751 crore. Also Read: Ola Electric, Kalyan Jewellers among 10 firms where promoters pledge increased in Q4 ( Disclaimer : Recommendations, suggestions, views and opinions given by the experts are their own. These do not represent the views of the Economic Times)
Yahoo
04-06-2025
- Business
- Yahoo
French ANSM authorises FineHeart's device trial for heart failure
The French National Agency for the Safety of Medicines and Health Products (ANSM) has authorised FineHeart to commence a nonrandomised first-in-human trial in the country to assess the FlowMaker device in heart failure patients. This prospective trial aims to evaluate the feasibility of device implantation alongside its preliminary clinical performance and safety. It comes after the initial implantations of the device at the Institute for Clinical and Experimental Medicine in Prague, Czech Republic, last year. The latest trial will be carried out across several cardiac surgery-specialising centres in France. According to the company, the fully implantable left ventricular assist device operates in synergy with the natural contraction of the heart. It is less invasive than current devices and is designed to enhance an individual's quality of life by preserving native cardiac function. Designed by French rhythmologists and electrophysiologists, the device claims to be the first implantable cardiac output accelerator. It adapts to the pathology's progression and can be removed with no risk to the patient. It does not necessitate aortic bypass. The device utilises less energy and operates without percutaneous connections to external batteries, as it is recharged through a transcutaneous energy transfer system, aiding in the elimination of infection risks. The implantation procedure is performed on a beating heart and typically lasts around 90 minutes. FineHeart said that the device offers a therapeutic option for over 200,000 individuals annually with advanced heart failure and those who are ineligible for existing treatments. FineHeart co-founder and CEO Arnaud Mascarell said: "ANSM approval marks a key strategic step for FineHeart. It validates the quality and solidity of our preclinical work and confirms the relevance of our initial clinical results. 'This green light paves the way for the acceleration of our clinical roadmap, with the ambition of responding to a major unmet medical need. It also sends a strong signal to the entire medtech ecosystem, and to all our partners and investors, whom we thank for their confidence.' In 2023, the company secured two patents from the China National Intellectual Property Administration for FlowMaker. "French ANSM authorises FineHeart's device trial for heart failure" was originally created and published by Medical Device Network, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio