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Press Release

Neurogene’s Gene Replacement Program is the Eighth Biopharmaceutical Company Program Facilitated by the Rett Syndrome Research Trust

Media Contact
Monica Coenraads
Chief Executive Officer
Rett Syndrome Research Trust
May 18, 2022

TRUMBULL, CT – The research and resource development efforts of the Rett Syndrome Research Trust (RSRT) have helped generate an eighth biopharmaceutical program that tackles the root cause of Rett syndrome. Neurogene, a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, today announced that it will be expanding the company’s development pipeline with the addition of NGN-401 for Rett syndrome. NGN-401 is a gene replacement approach that utilizes Neurogene’s novel EXACT gene regulation technology. Stuart Cobb, PhD, Chief Scientific Officer at Neurogene and Simons Fellow at the University of Edinburgh, presented preclinical efficacy and safety data for NGN-401 at the 25th Annual Meeting of the American Society for Gene and Cell Therapy. Gene replacement is one of six strategies championed by RSRT to target the root cause of Rett syndrome and is the strategy closest to the clinic.

Neurogene was founded in 2018 by Rachel McMinn, PhD. Dr. McMinn, Neurogene’s Chief Executive Officer, was inspired by her brother, who lives with a rare, debilitating neurological condition. She dedicated her early career to science and worked as a biotechnology equity analyst for over 13 years before founding Neurogene. RSRT has collaborated with both Neurogene and the University of Edinburgh for a number of years. Over the past decade, RSRT has provided almost $3 million to the University of Edinburgh to support Dr. Cobb’s research on gene replacement and RNA modification approaches to cure Rett syndrome.

“We are delighted to announce our development program in Rett syndrome, which employs Neurogene’s novel EXACT gene regulation technology that enables NGN-401 to deliver therapeutic levels of MECP2 without causing toxicity in preclinical studies. We recognize RSRT for its significant efforts and collaboration, championing innovation to address the limitations of conventional gene therapy,” said Neurogene CEO Dr. Rachel McMinn.

Rett syndrome, caused by mutations in the MECP2 gene located on the X chromosome, affects 350,000 individuals worldwide, most of whom are female. Gene replacement, which delivers a healthy gene to compensate for the defective one, has the potential to cure or significantly reduce the devastating symptoms of Rett, which include losing acquired skills such as walking, talking, and hand use, and symptoms such as scoliosis, extreme anxiety, tremors, seizures, hyperventilation, and digestive problems. Most people with Rett syndrome live into adulthood, requiring round-the-clock care.

“We are grateful to RSRT for their substantial funding to the University of Edinburgh, supporting key scientific discoveries in Rett syndrome,” said Dr. Cobb. “Moreover, RSRT facilitated my connection to Neurogene, which has led to increased resourcing and scientific collaboration, and, ultimately, the acceleration of our research into a potential treatment for Rett syndrome.”

For almost two decades, Dr. Cobb has studied the physiology of the Rett brain. In 2007 he co-authored, with Sir Professor Adrian Bird, the landmark paper that described the reversibility of Rett syndrome in mice. As a founding member of RSRT’s Gene Therapy Consortium, a collaboration among experts in gene therapy and Rett syndrome, Dr. Cobb helped generate discoveries that led to the advancement of Rett syndrome gene replacement programs at three biopharmaceutical companies, including Taysha Gene Therapies and AveXis, in addition to Neurogene.

“RSRT is dedicated to finding a cure for Rett syndrome, and we are excited to see the science continue to progress toward that vision,” said RSRT CEO Monica Coenraads, “My colleagues and I will do everything in our power to help the Rett program at Neurogene succeed. We are eager to extend discoveries from our efforts to identify Rett syndrome biomarkers and objective outcome measures, along with data from the Rett Syndrome Global Registry and digital natural history study, to Neurogene and other partner companies to accelerate their Rett programs.”

In September 2021, amidst a challenging regulatory environment for gene replacement clinical trials, Neurogene received clearance from the US Food and Drug Administration (FDA) to initiate a gene therapy clinical trial for children affected by CLN5 Batten disease, a rare and fatal neurogenerative disorder for which there are no approved disease-modifying therapies.

“The FDA is carefully scrutinizing gene replacement programs, and the fact that Neurogene achieved clearance to move forward with a clinical trial to treat Batten disease, a disorder that, like Rett syndrome, has no genetic-based therapies, is a very positive milestone,” said Randy Carpenter, RSRT’s Chief Medical Officer. “I have total confidence in the Neurogene team and look forward to supporting them in their efforts to advance NGN-401.”

Neurogene’s Rett program is the eighth genetic-based biopharmaceutical company program to be either incubated by RSRT or advanced by access to RSRT resources, such as its biorepository. These programs include RNA editing programs at Shape Therapeutics, Vico Therapeutics, and Wave Life Sciences; MECP2 reactivation programs at Alcyone Therapeutics and Herophilus; a gene editing program at Beam Therapeutics; and a gene replacement program at Taysha Gene Therapies.

“I’m profoundly grateful to our loyal and generous donors and to the families affected by Rett syndrome who take action and fundraise,” said Coenraads. “Thanks is also due to international Rett organizations that have generously supported RSRT, especially Reverse Rett in the UK, the Rett Syndrome Research & Treatment Association in Israel, and Rett Syndrome Deutschland. The addition of Neurogene’s program to the growing number of therapeutics in development is proof that the energy, dedication, and efforts of these families and organizations are making an enormous impact.”

RSRT is a nonprofit organization with a highly personal and urgent mission: achieving a cure for Rett syndrome and related disorders caused by defects in the MECP2 gene. Since its founding in 2008, RSRT has awarded $64 million, more than any other Rett organization in the world, to leading scientists pursuing targeted research on Rett. RSRT funds and spearheads global scientific and clinical activities advancing the most promising curative approaches. To date, every biopharmaceutical company pursuing a cure for Rett syndrome is doing so because they leveraged discoveries and resources incubated with RSRT funding. A highly efficient nonprofit, RSRT has spent an average of 95 percent of every dollar on its research program.

Neurogene’s mission is to turn devastating neurological diseases into treatable conditions to improve the lives of patients and families impacted by these rare diseases. Neurogene is working on cutting edge science to address limitations of conventional gene therapy in CNS disorders by selecting a delivery approach to maximize distribution to target tissues, designing products to maximize potency and purity for an optimized efficacy and safety profile, and using the company’s novel EXACT gene regulation platform technology for complex disorders to deliver therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. For more information, visit