Cockayne Syndrome

This collaboration marks a significant advancement towards finding a treatment for this fatal autosomal recessive disorder. The initiative is significantly supported by the Riaan Research Initiative, a nonprofit patient-advocacy organization, which has provided the largest known donation towards Cockayne syndrome research. This partnership between Andelyn and UMass Chan Medical School, bolstered by the Riaan Research Initiative’s contribution, brings clinical trials for this devastating condition closer to reality. Advancing gene therapy Miguel Sena-Esteves associate professor of neurology and director of the Translational Institute for Molecular Therapeutics at UMass Chan Medical School, expressed his optimism about the collaboration: “Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction. “Developing a gene therapy for fatal pediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome.” Cockayne syndrome is a rare genetic disorder characterized by developmental and neurological degeneration. Currently, there are no effective treatments, making this collaboration a beacon of hope for affected families. Andelyn’s role and expertise Matt Niloff, chief commercial officer of Andelyn, emphasized the significance of their involvement in this project. “We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome. With more and more organizations across the industry adopting our data-driven and configurable AAV Curator™ Platform, we are excited to add UMass as an important partner leveraging the platform to drive the acceleration of the Cockayne syndrome program.” googletag.cmd.push(function () { googletag.display('text-ad1'); }); Andelyn Biosciences is renowned for its high-quality and scalable end-to-end development and manufacturing capabilities. Their expertise will be pivotal in industrializing the production process for the AAV9-CSA vector, ensuring consistent and reliable manufacturing of this gene therapy. Their facilities in Columbus, Ohio, are equipped to meet the rigorous demands of such innovative treatments. Looking ahead The collaboration between Andelyn Biosciences and UMass Chan Medical School represents a significant milestone in the fight against Cockayne syndrome. By harnessing Andelyn’s advanced manufacturing capabilities and UMass Chan’s pioneering research, the partnership aims to accelerate the development of a viable gene therapy for this challenging disorder. The successful production of clinical-grade AAV9-CSA vectors will pave the way for first-in-human clinical trials, offering hope for a treatment that could significantly improve the lives of those affected by Cockayne syndrome. With ongoing support from organizations like the Riaan Research Initiative, the path forward for gene therapy treatments for rare diseases looks promising. This partnership exemplifies how collaborative efforts and innovative technologies can drive progress in the field of gene therapy, bringing life-changing treatments closer to those in need.

COLUMBUS, Ohio, July 10, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by UMass Chan Medical School to use its suspension AAV Curator™ Platform to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome. The nonprofit patient-advocacy organization, Riaan Research Initiative, has provided the largest known donation ever made toward the development of a treatment for Cockayne syndrome. The gift from Riaan Research Initiative and the partnership with Andelyn brings the start of clinical trials for the fatal autosomal recessive disorder one step closer. Andelyn's extensive experience in gene therapy development and manufacturing will be leveraged to industrialize the production process and drive program success. Miguel Sena-Esteves, PhD, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics said, "Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction. Developing a gene therapy for fatal pediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome". Matt Niloff, Chief Commercial Officer of Andelyn, adds, "We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome. With more and more organizations across the industry adopting our data-driven and configurable AAV Curator™ Platform, we are excited to add UMass as an important partner leveraging the platform to drive the acceleration of the Cockayne syndrome program." With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn continues to partner with premier innovator organizations to enable the progression of life-altering cell and gene therapies to cure rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn accelerates programs and delivers high quality products by manufacturing product on its configurable, data-driven AAV Curator™ Platform, or through a tech transfer in of established client programs. Capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A rigorous quality system, regulatory support and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit andelynbio.com. About UMass Chan Medical School UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine; the Morningside Graduate School of Biomedical Sciences; the Tan Chingfen Graduate School of Nursing; ForHealth Consulting of UMass Chan Medical School, a public service health care consulting division; and MassBiologics, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. At UMass Chan, we are advancing together to improve the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. UMass Chan has built a reputation as a world-class destination for biomedical research, with more than $300 million in annual funding and more than 500 active clinical trials and as a leader in primary care education, perennially ranked in the top 10 percent of medical schools for primary care by U.S. News and World Report. In 2021, the Medical School received a $175 million donation from The Morningside Foundation and was renamed UMass Chan Medical School. Learn more at . SOURCE Andelyn Biosciences

Drugs that selectively kill senescent cells may benefit otherwise healthy older women but are not a 'one-size-fits-all' remedy, Mayo Clinic researchers have found. Specifically, these drugs may only benefit people with a high number of senescent cells, according to findings publishing July 2 in Nature Medicine. Drugs that selectively kill senescent cells may benefit otherwise healthy older women but are not a "one-size-fits-all" remedy, Mayo Clinic researchers have found. Specifically, these drugs may only benefit people with a high number of senescent cells, according to findings publishing July 2 in Nature Medicine. Senescent cells are malfunctioning cells in the body that lapse into a state of dormancy. These cells, also known as "zombie cells," can't divide but can drive chronic inflammation and tissue dysfunction linked to aging and chronic diseases. Senolytic drugs clear tissues of senescent cells. In the 20-week, phase 2 randomized controlled trial, 60 healthy women past menopause intermittently received a senolytic combination composed of FDA-approved dasatinib and quercetin, a natural product found in some foods. It is the first randomized controlled trial of intermittent senolytic treatment in healthy aging women, and the investigators used bone metabolism as a marker for efficacy. Researchers found that this combination, known as D+Q, had beneficial effects on bone formation but did not reduce bone resorption or the breakdown and removal of bone tissue. Furthermore, D+Q mainly benefited people with evidence of a high number of senescent cells. This group had more robust increases in bone formation, decreases in bone resorption, and an increase in bone mineral density at the wrist. "Our findings argue against what many people are already doing -- using commercial products like quercetin or related compounds like fisetin that may show some senolytic properties," says senior author Sundeep Khosla, M.D., an endocrinologist at Mayo Clinic in Rochester, Minnesota. "They're using them as anti-aging agents without knowing if they have high enough senescent cell numbers to benefit, or what dose or dosing regimen is needed to be effective yet safe." Dr. Khosla says more research is needed to better identify people who may benefit from senolytic treatments and to develop more specific and potent senolytic drugs that may show efficacy in more people. People who have experienced "accelerated aging" -- such as cancer survivors after chemotherapy, or those with progeroid syndromes -- may have increased numbers of senescent cells. Besides their application to aging, senolytic drugs may be useful against certain diseases, such as idiopathic pulmonary fibrosis, dementia, diabetes, heart disease and others, Dr. Khosla says. However, these drugs will likely need to be customized according to their potency and the amounts of senescent cells in the diseased tissues. The study was supported by National Institutes of Health grant nos. R21 AG065868, P01 AG062413, R01 AG 076515, R01 DK128552, R01 AG055529, R37 AG13925 and R33 AG61456. Co-authors are Joshua Farr, Ph.D., Elizabeth Atkinson, Sara Achenbach, Tammie Volkman, Amanda Tweed, Stephanie Vos, Ming Ruan, Jad Sfeir, M.D., Matthew Drake, M.D., Ph.D., Dominik Saul, M.D., Madison Doolittle, Ph.D., Irina Bancos, M.D., Kai Yu, M.D., Tamara Tchkonia, Ph.D., Nathan LeBrasseur, Ph.D., James Kirkland, M.D., Ph.D., and David Monroe, Ph.D. Drs. LeBrasseur, Tchkonia and Kirkland have financial interests related to this research, including Mayo Clinic patents and pending patents covering senolytic drugs and their uses. The remaining authors declare no competing interests.