Children's Hospital of Orange County

IRVINE, Calif., Aug. 19, 2024 /PRNewswire/ -- University Lab Partners (ULP) is proud to announce the winners of the 4th annual Pitch. Launch. Grow. competition, a hallmark event that continues to spotlight innovation in life sciences, medical technology, and pediatrics. This year's competition attracted over 40 applications, with startups competing for recognition across three specialized tracks. Continue Reading The winners in the Life Science and Medical Technology tracks each receive a one-year sponsored lab bench at University Lab Partners, the premier wet lab incubator in Irvine, California. The Pediatric track winner will not only receive the lab bench sponsorship but also a $2,500 prize from the Consortium for Technology & Innovation in Pediatrics (CTIP)—an FDA-funded pediatric device accelerator—and a one-hour virtual mentorship session with Dr. Terence Sanger, MD, PhD, Chief Scientific Officer at CHOC Research. ULP's Pitch. Launch. Grow. competition spotlights innovation in life sciences, medical technology, and pediatrics. Post this Dr. Sanger commended all participants, stating, "We extend our heartfelt gratitude to every founder in the Pediatric Device track. Your dedication and innovative spirit are exactly what the Pitch. Launch. Grow. competition aims to foster." Pitch. Launch. Grow. 2024 Winners: Life Science - Hybrid Biotherapeutics Inc. & Cellecho Biotech Medical Technology - Syntr Health Technologies, Inc. & iPill Dispenser Pediatric - Remmie Health ULP's life science startups are provided with unparalleled access to professional mentoring, consulting with Experts-in-Residence, networking events, and unique access to UCI's technical core facilities. Since its inception in 2019, ULP has incubated 64 companies that have collectively raised over $535 million, successfully graduating eight startups into the broader market. Konstantin Kazarian, CEO at Hybrid-Bio, shared, "As developers of a new solid tumor cell therapy, we're thrilled by the interest the competition brought us. This recognition means a lot to Hybrid Biotherapeutics, and we look forward to collaborating with ULP and utilizing their state-of-the-art laboratory facilities." The 2024 Pitch. Launch. Grow. winners were selected by a distinguished panel of judges, representing leading organizations including Johnson & Johnson, Wilson Sonsini Goodrich & Rosati, CHOC, General Inception, Woodgreen Ventures, Xinetix, and D2M Venture Development. Pitch. Launch. Grow. is made possible thanks to the generous sponsorship of Sunstone Management, Insperity, HATCHspaces, NexCore Group, and Wilson Sonsini Goodrich and Rosati. For more information, please visit About University Lab Partners: University Lab Partners (ULP) is a premier, nonprofit, wet lab incubator located in Orange County, CA. ULP operates 2 facilities in Irvine and Aliso Viejo, CA. ULP offers highly equipped wet lab facilities along with the benefits of accelerator services and peer-to-peer interactions among a life science-focused entrepreneurial community. SOURCE University Lab Partners

Alcyone's ThecaFlex is an implantable medical device under investigation for routine subcutaneous administration of therapeutics to the cerebrospinal fluid (CSF). The first stage of the pivotal IDE PIERRE trial has been completed. 10 spinal muscular atrophy (SMA) patients have undergone ThecaFlex implantation and have received nusinersen through the device. No device-related adverse events, including infections, were observed in the 30 days after implantation. FDA has agreed to an additional 80-SMA patient, 30-center, enrollment after review of the clinical data by Data Monitoring Safety Committee of first 10 SMA patients implanted. LOWELL, Mass., July 31, 2024 /PRNewswire/ -- Alcyone Therapeutics Inc. (Alcyone), a clinical-stage biotechnology company pioneering next-generation precision genetic therapies and precision delivery solutions of therapies for complex neurological conditions, announced today that the U.S. Food and Drug Administration (FDA) has provided approval to continue enrollment of the PIERRE study () to evaluate the safety and effectiveness of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system for chronic intrathecal access, CSF aspiration, and delivery of SPINRAZA® (nusinersen) in SMA patients as an alternative to repeat lumbar puncture (LP). As previously announced, the PIERRE IDE trial was designed to enroll up to 90 SMA patients with a minimum age of 3 years old and be conducted in two stages. The first stage, consisting of 10 SMA patients enrolled, implanted, infused with nusinersen and followed for 30 days, is complete. During the first stage of the trial, all attempts to aspirate and infuse through the ThecaFlex port were successful, and no infection or device-related adverse events were observed. Based on a review of the data from the first stage, the FDA provided approval to continue enrollment of an additional 80 SMA patients in up to 30 centers across the U.S. and Europe. Based on experience among the first 10 SMA patients enrolled in the trial, the observed duration for implantation of ThecaFlex was between one and two hours, depending on spinal complexity of the patient, with most SMA patients discharged from the hospital within 24 hours of implantation. Following implantation, the infusion procedure was typically performed in a non-specialized setting (i.e., a standard exam room) with an observed infusion procedure duration of less than 30 minutes with the actual nusinersen subcutaneous infusion time taking less than 10 minutes. "As we approach the next stage of the PIERRE IDE study, we will continue to evaluate the safety and efficacy of the ThecaFlex system to help patients with spinal muscular atrophy receive the SPINRAZA® (nusinersen) therapy," says Dr. Scellig Stone, M.D., Ph.D., FRCSC the Boston Children's Hospital primary investigator for the PIERRE study. "The initial data and patient feedback are optimistic and a key first step toward developing a less invasive, more accessible procedure that may improve the overall dosing experience for SMA patients and may help optimize hospital resources." "We were pleased to receive the recommendation from the independent data monitoring safety committee to continue enrollment without any changes to the PIERRE study protocol as well as the FDA's subsequent approval to continue enrollment," said Dr. Kathrin Meyer, Ph.D., Alcyone's Chief Scientific Officer and Head of Research & Development. "This represents a tangible step toward our goal of substantially improving treatment experience for patients requiring repeat intrathecal delivery of medications for the treatment of neurological disorders." Potential clinical benefits of the ThecaFlex DRx System are currently being evaluated in the PIERRE study and may include: (a) enabling routine subcutaneous administration of ASO (antisense oligonucleotide) therapies to the cerebrospinal fluid, and (b) reducing the need for anesthesia and radiation exposure compared to repeat LP for SPINRAZA infusion in SMA subjects. "For those who currently require anesthesia and radiographic guidance for delivery of nusinersen, which exposes them to radiation, ThecaFlex could shift administration from a specialized visit to an exam room setting," said Dr. Michael Muhonen, M.D. at the Children's Hospital of Orange County in California. "I look forward to the continued enrollment of the study and future learnings from the patient therapy administration experience with this innovative device." Beyond this trial, if ThecaFlex is approved for chronic bolus intrathecal administration of drugs without indication restrictions, it may increase accessibility for people suffering from other neurological disorders that need repeat intrathecal drug delivery. It has the potential to be the first implantable device designed to enable routine subcutaneous access for the delivery of ASO therapies. The PIERRE pivotal IDE trial is part of a collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone previously announced in January 2023. Additional details can be found at . About The ThecaFlex DRx™ System The ThecaFlex DRx System (ThecaFlex), a technology within Alcyone's Falcon™ Delivery Platform, is an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies by IT bolus administration. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with a challenging spinal anatomy or those requiring multiple anesthesia and radiation exposures for repeat LPs, or for patients for whom the treating physician determines implantation of ThecaFlex is otherwise in the patient's best interests. ThecaFlex has received a CE Mark in Europe and IDE (Investigational Device Exemption) from FDA to conduct a clinical investigation but has not yet been approved for commercial use by FDA. In addition, ThecaFlex has received Breakthrough Device Designation from FDA. For more information, visit . About Alcyone Therapeutics Alcyone Therapeutics is a clinical-stage biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon™, the Company's proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebrospinal fluid (CSF) dynamics, computational modeling, bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute (AWRI) at Nationwide Children's Hospital. This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone's lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome. For more information, visit . About SPINRAZA® (nusinersen) SPINRAZA is approved to treat infants, children, and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.1 SPINRAZA is an antisense oligonucleotide (ASO) that targets an underlying cause of motor neuron loss in SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.2 SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated for up to eight years, combined with unsurpassed real-world experience.3 The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). SHINE and ongoing NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country's product website. References: Based on commercial patients, early access patients, and clinical trial participants through March 31, 2022. SPINRAZA U.S. Prescribing Information available at: . Accessed: October 2022. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA. SOURCE Alcyone Therapeutics

A gift from Sherry and John Phelan will advance whole-child wellness by providing lifestyle, dietary and psychological support ORANGE, Calif., July 30, 2024 /PRNewswire/ -- A generous gift will help establish an innovative integrative gastroenterology multidisciplinary clinic and a culinary medicine program at Children's Hospital of Orange County (CHOC), enhancing the treatment of children with gastroenterological conditions by adding evidence-based integrative care aided by food and nutrition. Thanks to the generosity of Sherry and John Phelan, the program will provide patients with coordinated, holistic care and the dietary, emotional and psychosocial support they need to cope with their symptoms and ultimately find relief, health and healing. These additions will incorporate multidisciplinary specialties such as psychology, nutrition and complementary medicine into CHOC's already robust gastroenterology program. "We are incredibly grateful for the visionary support of Sherry and John Phelan," said Dr. Ashish Chogle, who is medical director of gastroenterology at CHOC and will lead the new programs. "Their generous gift underscores their continued commitment to holistic healthcare and dedication to making a difference in the lives of innumerable children. Their generosity will help CHOC emerge as a true leader in pediatric integrative healthcare. Our care model will deliver better outcomes and an elevated family experience. Ultimately, we hope to influence public health strategies and foster widespread acceptance of holistic health practices, including using food as medicine in pediatric care." The Phelans' generous gift will allow the integrative gastroenterology clinic to be held weekly to support patients with various conditions, including chronic abdominal pain, irritable bowel syndrome, gastrointestinal motility disorders, gastroesophageal reflux disease (GERD), constipation, and nutrition- and obesity-related issues. Thanks to the gift, numerous patients will benefit from a pediatric psychologist trained in cognitive behavior therapy (CBT) and hypnotherapy; a dedicated pediatric dietician; and other clinicians trained in various integrative treatment modalities. Additionally aligned with the philosophy that food is medicine, the generous gift will establish the foundation for a culinary medicine program to support CHOC gastroenterology patients. The program will include educational workshops led by medical chefs. Longtime supporters of CHOC, John and Sherry Phelan have previously supported the healthcare system's mental health, child life, neonatology and research programs. "We are excited to support this comprehensive program at CHOC and have all the confidence in the team, which is rapidly moving this concept into action with a brilliant clinician and staff and plan in place," said Sherry Phelan, PhD, an organizational psychologist and artist who integrates her psychology training into her art. Dr. Phelan also chairs the Advisory Board of UCI's Susan Samueli Integrative Health Institute. About CHOC: CHOC, a pediatric healthcare system based in Orange County, California, is committed to being a leading destination for children's health by providing exceptional and innovative care. Our growing community includes two state-of-the-art hospitals in Orange and Mission Viejo and a regional network of primary and specialty care clinics serving children and families in four counties. CHOC offers several clinical programs of excellence providing the highest levels of care for the most serious pediatric illnesses and injuries, both physical and mental. Our research and innovation institutes are focused on translating real patient needs into real-world treatments so every child can live the healthiest and happiest life possible. To learn more, visit . SOURCE Children’s Hospital of Orange County (CHOC)