Spark Therapeutics, Inc.

Karl Diesseroth of Stanford University, Peter Hegemann of Berlin’s Humboldt University and Georg Nagel of the University of Würzburg will share the winnings for the 2026 Nobel Prize in Physiology or Medicine. Credit: AntonSAN / Shutterstock.com. A trio of German and American scientists have won this year’s Nobel Prize in Physiology or Medicine for their discovery of a new brain activity-mapping and nerve signal-controlling method – giving credence to an approach pharma and biotech are currently exploring to treat several ophthalmic conditions. Through this landmark win, Karl Diesseroth of Stanford University, Peter Hegemann of Berlin’s Humboldt University and Georg Nagel of the University of Würzburg will split a prize pot of SKr 12m ($1.19m), which the Nobel committee is awarding to the scientists for their work on discovering and developing the field of optogenetics. At the core of the optogenetic approach is channelrhodopsin, an ion channel protein found in single-celled algae that opens upon exposure to blue light. After channelrhodopsin’s initial discovery in the early 2000s, the three Nobel Prize winners realised that the protein could be used to map the activity of nerve cells and how they shape the creation of memories, feelings and behaviours in the living brain. This gave rise to optogenetics as we know it today, which is being used to guide research into neurological and psychiatric disorders, among other things. According to Per Svenningsson, chair of the Nobel Committee for Physiology or Medicine, optogenetics provides scientists with “opportunities for mapping the brain that we could once only dream of”. However, drugmakers are now betting that the approach will prove useful in the field of ophthalmology. This has led several biotech and pharma companies to expllore the promise of optogenetics in a clinical setting, with several drugs currently in development as vision-restoring treatments for patients with certain degenerative eye conditions. The most advanced medicine harnessing this technique is Nanoscope Therapeutics’ optogenetic gene-agnostic therapy, Mogenry (sonpiretigene isteparvovec), which is designed to restore vision in patients with severe retinal degeneration. Nanoscope created the one-time medicine to introduce a light-sensitive opsin protein to intact visual signal-transmitting cells in the eye, allowing them to better respond to light and improve vision despite photoreceptor death. Thus far, the drug has already shown late-stage promise in both retinitis pigmentosa (RP) and Stargardt disease , with US regulators having accepted the biologics license application (BLA) for MCO-010 in RP back in September. However, Nanoscope is not the only company looking to bring an optogenetic drug to patients, as companies like GenSight Biologics and Ray Therapeutics are also evaluating their gene therapies, GS030 and RTx-01, in RP clinical trials. Currently, Roche-owned Spark Therapeutics and Novartis’ Luxturna (voretigene neparvovec) is the only vision-restoring therapy approved by the US Food and Drug Administration (FDA) for the treatment of RP. Analysts at GlobalData, the parent company of Pharmaceutical Technology , estimate that Luxturna’s sales will approach the $400m mark in 2032.

Accomplished Biopharma Executive Joins Tortugas to Help Lead Continued Growth FRAMINGHAM, Mass.--(BUSINESS WIRE)-- Tortugas Neuroscience (“Tortugas”), a clinical-stage biotech company developing medicines for central nervous system (CNS) disorders, today announced the appointment of Ramzi Benamar as Chief Financial Officer (CFO). Benamar is a biotechnology executive with experience in capital formation, corporate strategy, M&A, operational scaling, and commercial launch preparation. He has participated in and led these activities across multiple life sciences companies. “Ramzi brings meaningful capabilities and an understanding of the life sciences industry. He has had a documented and successful track record of helping biotechnology companies navigate important stages of growth,” said Jeff Jonas, M.D., Chief Executive Officer of Tortugas. “His perspective and expertise will be critical as we continue to advance our pipeline and execute on our long-term corporate and financial strategy.” Launched in April 2026, Tortugas is advancing several product candidates selected for their differentiated pharmacology, as well as their clinical and translational data supporting efficient clinical development in large-market indications with well-defined development and regulatory pathways. The company has a clinical stage pipeline of four assets, featuring potential treatments for schizophrenia, tinnitus, focal epilepsy, reversible encephalopathies, and other CNS indications. These programs, in-licensed from Jiangsu Hansoh Pharmaceutical Group Ltd. and Eisai Co., Ltd., are comprised of small molecule new chemical entities (NCEs) with differentiated mechanisms of action. “Tortugas has assembled a differentiated, clinical-stage CNS pipeline and a leadership team with extensive experience developing successful new medicines in CNS,” said Mr. Benamar. “I am excited to join the company at this important stage and to help build the financial and corporate strategy needed to advance these promising programs.” Benamar previously served as CFO of the clinical-stage biotech Bellus Health, where he helped lead the company through its $2.0 billion acquisition by GSK. Most recently, he was CFO of Apnimed, where he was focused on capital allocation and commercialization planning for the company. Previously, Benamar served as CFO at DBV Technologies and Elucida Oncology, in addition to holding senior leadership roles at major biotechnology and pharmaceutical companies such as Spark Therapeutics, Johnson & Johnson, Shire, and Merck. Benamar holds a Master of Business Administration and a Bachelor of Business Administration from Temple University as well as a master’s degree from the University of the Sciences in Philadelphia. About Tortugas Neuroscience Tortugas Neuroscience is a clinical-stage CNS-focused biotech company dedicated to developing medicines that address diseases of the brain. With a strategic focus on clinically validated mechanisms of action with well-defined development and regulatory pathways, Tortugas Neuroscience is committed to creating therapies with meaningful impact for patients worldwide. For more information, please visit: Contacts Media Contact: Rachel Ford Hutman Rachel@fordhutmanmedia.com

MILAN, Sept. 01, 2026 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company pioneering therapies for inherited retinal diseases (IRDs) with clinical programs in Usher syndrome type 1B and Stargardt disease enabled by its proprietary dual-AAV platform designed to overcome the AAV cargo capacity limit, today announced the appointment of Megan Zoschg Canniere, Pharm.D. , as Chief Regulatory & Quality officer. Dr. Zoschg Canniere joins the Company’s leadership team to support the continued advancement of AAVantgarde’s clinical-stage pipeline and regulatory strategy and will report to Dr. Natalia Misciattelli, Chief Executive Officer. “Megan brings exceptional regulatory and drug development expertise, with deep experience in gene therapy and rare diseases, as well as an outstanding track record of successfully advancing innovative programs through complex regulatory pathways,” said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde . “As AAVantgarde progresses its clinical programs toward late-stage clinical development, her extensive experience in regulatory strategy, health authority interactions and clinical development will be invaluable. We are delighted to welcome Megan to our leadership team at this important stage of the Company’s growth.” “I am excited to join AAVantgarde at such an important stage in the Company’s development,” said Megan Zoschg Canniere, Pharm.D., Chief Regulatory & Quality officer at AAVantgarde . “AAVantgarde has built a strong clinical pipeline addressing significant unmet needs in inherited retinal diseases, and I look forward to working with the team to advance these programs through clinical development and towards regulatory approval, ultimately bringing potentially transformative therapies to patients.” Prior to joining AAVantgarde, Ms. Zoschg Canniere spent five years at Spark Therapeutics (Roche Group), where she held senior leadership positions, most recently serving as Executive Vice President and Chief Development Officer. At Spark, she led clinical development and regulatory strategies across multiple programs and trials, including innovative gene therapy development approaches and interactions with health authorities across regions. Previously, Dr. Zoschg Canniere spent 15 years at Roche/Genentech, where she led global regulatory strategies and health authority interactions across neuroscience and rare disease programs, including gene therapy. Earlier in her career, she held regulatory leadership positions at Warner Chilcott, Roche and Johnson & Johnson Pharmaceutical Research & Development. She holds a Doctor of Pharmacy degree from the University of Pittsburgh. About AAVantgarde Bio AAVantgarde is a clinical stage, biotechnology company advancing best-in-class therapies for patients with inherited retinal diseases. The company’s lead programs target Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B, two severe, inherited retinal diseases with no approved treatments. With a strong foundation in translational science and a commitment to clinical excellence, AAVantgarde is working to bring transformative therapies to patients. For more information, please visit: Media Contact: Barnaby Pickering – Director, 59 North Communications Barnaby.Pickering@59north.bio