A promising new drug aimed at treating a debilitating neurological disorder that affects children is being manufactured here in Houston and is now under FDA clearance for investigation. This is another sign of a growing life sciences research and manufacturing infrastructure in the region.
Neurogene Inc., a clinical-stage pharmaceutical company, said this week that the FDA had approved an application for NGN-401 to treat Rett syndrome, a progressive neurological disorder that affects 1 in 10,000 female babies. said. The syndrome is caused by genetic mutations that can affect the brain and nervous system during early development, causing rapid regression of language, fine motor skills, and other symptoms after 18 months. There is no disease-modifying therapy for the syndrome, and doctors only manage symptoms.
Neurogene’s NGN-401 is the first investigational gene therapy to deliver the full-length human MECP2 gene, a specific gene that is mutated in patients diagnosed with Rett syndrome. This therapy uses the company’s proprietary His EXACT gene regulation technology, which can achieve desired levels of transgene expression within a narrow window critical for the treatment of Rett syndrome to avoid adverse side effects. According to the company, NGN-401 has undergone extensive preclinical trials, demonstrating strong efficacy. The treatment is performed at Neurogene’s GMP manufacturing facility in southwest Houston.
“NGN-401’s preclinical profile is very compelling and represents the strongest results to date in multiple animal models,” said Dr. Rachel McMinn, CEO and Founder of Neurogene. I’m here. “FDA approval of NGN-401 is an important milestone for Neurogene and the Rett syndrome community to transform a devastating neurological disorder into a treatable state and improve the lives of patients and families affected by these rare diseases. It underscores our commitment to
“Rett syndrome is a debilitating disease that has a devastating impact on children and their families, and there is no treatment to improve the disease,” said an assistant professor of pediatrics and neurology at Baylor College of Medicine and Texas Children’s Hospital. Neurologist Bernhard Suter, Ph.D. “Future clinical studies of NGN-401, whose mechanism of action is aimed at addressing the underlying causes of the disease, offer hope of improving the lives of people suffering from Rett syndrome.”
A 2022 study commissioned by the Greater Houston Partnership and developed by Newmark Consulting Group found that Houston’s industry-leading expertise in cell and gene therapy, biologics development, and molecular diagnostics will contribute to the growth of life sciences in the region. has emerged as a strong opportunity for
Houston ranks second in the nation for clinical trials, with more than 4,600 ongoing clinical trials, representing 15% of all ongoing clinical trials in the United States. The Houston-area agency received $864.1 million in National Institutes of Health (NIH) grants, an increase of 16.3% from 2020. Over the past five years, the region has received nearly $3.9 billion in funding from the NIH. That’s an average of $740.7 million annually. With the support of Texas Medical Center and world-class institutions that make up Houston’s significant patient population, Newmark’s research indicates that significant opportunities for commercial synergies exist with leading healthcare providers in the region. I understand.
Learn more about Houston’s life sciences sector.
