Spinal Cord Injuries

Boston biotech Axonis Therapeutics has raised a $115 million Series A to test whether its oral drug AXN-027 can help patients with focal epilepsy and diabetic neuropathic pain. The financing, disclosed Wednesday morning, will get the biotech into the clinic around the end of this year, or about the same time Ovid Therapeutics expects to begin human trials with a similar approach by way of an AstraZeneca platform it licensed. Both companies are looking at KCC2, a CNS chloride transporter that is key to electrical signaling in the brain. KCC2 allows for neuronal GABA signaling. The GABA pathway has been targeted by multiple drugmakers , including Sage’s postpartum depression medicine Zurzuvae , but biotechs have yet to enter the clinic with a focus on KCC2, CEO Joanna Stanicka told Endpoints News . “Often GABA treatments can induce global inhibition, which leads to this cognitive impairment and sedation often reported by patients,” Stanicka said, adding that focused inhibition should avoid those side effects. The 13-employee biotech also said Wednesday that it added Donald Manning as chief medical officer and Jeff Imbaro as operating chief. Manning previously held the same post at Travecta, Pacira BioSciences and Adynxx. Imbaro was in the same role at stroke-focused Elevian, and before that he was a founding member of Vertex-acquired Semma Therapeutics. Axonis was founded by spinal cord injury patient advocate and board observer Bob Yant about 15 years ago, but it didn’t become “operational” until 2019, Stanicka said. Prior to the Series A, Axonis had raised about $34 million in seed funding and about $9 million in government and foundation money, Stanicka said. Its investors include Cormorant Asset Management, venBio, Sofinnova Investments, Merck’s corporate venture arm and others. The startup anticipates the Series A will fund AXN-027 through Phase 2 proof-of-concept studies, she said.

RESEARCH TRIANGLE PARK, N.C, Oct. 22, 2024 /PRNewswire/ -- Dignify Therapeutics (Dignify), a leading developer of therapies for bladder and bowel dysfunction, has been selected as a participating company in the Praxis Spinal Cord Injury (SCI) Accelerate Program. SCI Accelerate is a commercialization program 'geared towards healthcare companies with a product ready to launch into market that will transform the lives of people living with SCI'. Under the SCI Accelerate program, Dignify and Praxis will focus on development of Dignify's drug programs to treat bladder and bowel disorders in people with SCI. Dr. Karl B. Thor, Chief Scientific Officer of Dignify, indicated; "The Praxis SCI Accelerate Program is critically important for Dignify to ensure that the clinical development program for our products provides the efficacy, safety, and convenience that are considered valuable and most important by SCI individuals for control of bowel and bladder dysfunction. The selection of Dignify to join the Praxis SCI community confirms the unmet medical need for our therapeutics." Arushi Raina, Praxis Director of Commercialization, stated "The Praxis team was impressed by the Dignify team's approach and excited by the possibility of a bowel and bladder treatment that could be used in an accessible way to transform daily living." About Dignify Therapeutics Founded by a team of serial entrepreneurs who are internationally recognized scientists and clinicians, Dignify's mission is to provide safe, effective, practical and convenient, "on-demand" pharmaceutical agents to treat bladder and bowel voiding dysfunctions. The company has been funded by RA Capital Management, Eshelman Ventures, the North Carolina Biotechnology Center, NIDDK, NINDS, NICHD, NIA, US Defense Department, and the One NC Small Business Program. Dignify is located at the First Flight Venture Center in Research Triangle Park, North Carolina. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. About Praxis Praxis is a Canadian-based not-for-profit organization that leads global collaboration in SCI research, innovation and care. We accelerate the translation of discoveries and best practices into improved treatments for people with spinal cord injuries. Our work is driven by the priorities of people with SCI and led by our four teams — PLEX (people with lived experience), Cure, Care and Commercialization. PRESS CONTACT Dignify Therapeutics Anthony DiTonno, CEO 2 Davis Drive Research Triangle Park, NC 27709 [email protected] dignifytherapeutics.com SOURCE Dignify Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Gene Therapy Automatically Converts Omega-6 to Omega-3 Fatty Acids in the Body ST. LOUIS, Oct. 16, 2024 /PRNewswire/ -- According to the Centers for Disease Control, nearly 20% of children and teens are considered obese. Research shows it can have a dramatic impact on a variety of health conditions, including arthritis, heart conditions and other metabolic problems, and the American Academy of Pediatrics now recommends early and intensive treatment to combat obesity. Over the last four years, Shriners Children's St. Louis researchers have been working to develop a new way to prevent the effects of childhood obesity. Using gene therapy, Shriners Children's St. Louis Director of Research Dr. Farshid Guilak and Senior Scientist Dr. Ruhang Tang have discovered a revolutionary new way to convert harmful fatty acids into beneficial ones, making it possible for children battling obesity to lower the risk of other health problems, particularly arthritis. "We've learned that the number one preventable risk factor for arthritis in children is being overweight," said Dr. Guilak. "Our typical response is simply telling kids to lose weight, without addressing the larger issue. Childhood obesity has become an epidemic, and as a result, arthritis is becoming increasingly common in children. One of our key findings is that it's not just the obesity itself that's harmful, but also the diet. The types of fatty acids children consume play a significant role in a child's weight gain. And once children are obese, it opens the door for other significant health problems that can be hard to reverse." Dr. Tang said their gene therapy technique involves utilizing an inactive virus, adeno-associated virus (AAV), to deliver the gene for a specific enzyme into cells. The new gene therapy automatically converts highly inflammatory Omega-6 fatty acids to Omega-3 fatty acids, which are better for the body's metabolic health. Dr. Guilak said Omega-6 acids, which come from fatty foods and vegetable oil such as in fried foods, tend to promote inflammation and can lead to health issues such as arthritis, heart disease, and metabolic problems. Omega-3s, as found in fish and certain nuts, can support a healthier metabolic profile by improving insulin sensitivity, promoting fat metabolism, and reducing inflammation and the risk of obesity-related diseases. Dr. Natalia Harasymowicz, co-lead of the study, said that their gene therapy was also able to lower the number of pro-inflammatory cells that are crucial in promoting obesity-related inflammation and arthritis. In this study, the researchers studied a type of arthritis that is very common in children, termed "post-traumatic arthritis", caused by injury to the knee such as a tear in the meniscus tissue that serves as a stabilizer and cushion in the knee. The team found that one injection of the gene therapy significantly decreased the effects of a high-fat, omega-6-rich diet on overall metabolic health and knee arthritis. Of particular interest was the finding that diet-induced obesity led to premature "aging" in these mice, which was prevented by gene therapy. "What we are observing is that obesity results in old knees in young patients", said Dr. Guilak. "The modern diet, especially in the United States, tends to be high in Omega-6s and low in Omega-3s, which can lead to an imbalance, especially for kids. The gene therapy we developed will automatically convert omega-6 to omega-3 fatty acids in your body, effectively turning bad fats into good fats. Arthritis can be a very painful and debilitating condition for children so we're hopeful that this research will help reduce the risk of it developing and make treatments more effective. The implications for how this research might be used in the future are vast but we're excited about the potential for it to help kids lead healthier lives." Dr. Guilak is also the Mildred B. Simon Professor of Orthopaedic Surgery at Washington University in St. Louis and co-Director of the Washington University Center of Regenerative Medicine. His team is working with the Food and Drug Administration (FDA) to head into clinical trials in the next 3-5 years. This research was supported by Shriners Children's and the National Institutes of Health. Ruhang Tang, Natalia S. Harasymowicz, Chia-Lung Wu, Yun-Rak Choi, Kristin Lenz, Sara J. Oswald, Farshid Guilak. Gene therapy for fat-1 prevents obesity-induced metabolic dysfunction, cellular senescence, and osteoarthritis. Proceedings of the National Academy of Sciences USA, Oct. 14, 2024. About Shriners Children's Shriners Children's improves the lives of children by providing pediatric specialty care, conducting innovative research, and offering outstanding education programs for medical professionals. Children with orthopedic conditions, burns, spinal cord injuries, and cleft lip and palate are eligible for care, regardless of the families' ability to pay, and receive all care and services in a compassionate, family-centered environment. For more information, please visit shrinerschildrens.org. SOURCE Shriners Children's WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED