Latest news with #JeffSperring
Business Wire
15-05-2025
- Health
- Business Wire
FDA Grants Regenerative Medicine Advanced Therapy Designation for BrainChild Bio's B7-H3 CAR T-cell Therapy for Incurable Pediatric Brain Tumors
SEATTLE & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), today announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. The use of a regenerative medicine, specifically a CAR T-cell therapy, offers the potential to overcome barriers for other drug modalities to be effective in addressing DIPG, including the precarious location of the DIPG tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains intact during tumor progression. BrainChild Bio has designed BCB-276 to be administered by locoregional delivery of targeted CAR T-cells directly into the cerebrospinal fluid, permitting infused CAR T-cells to directly access the tumor bed, using an indwelling reservoir-catheter device. This approach to administering an autologous B7-H3 CAR T-cell therapy has been successfully implemented and resulted in the promising overall survival benefit in patients with brain tumors observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio's academic partner, Seattle Children's Research Institute, and recently published in Nature Medicine. 'We are very pleased to now also receive RMAT designation, less than one month after being granted Breakthrough Therapy designation from FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG. Receiving designations from two independent reviews within FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG,' stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. 'Our team is keenly focused on initiating the pivotal Phase 2 trial by the end of this year and look forward to continuing to work with the FDA on an accelerated path forward to bring potential new CAR-T treatments for CNS brain tumors in children and adults.' FDA grants RMAT designation to investigational regenerative medicine therapies, including cell therapies, that are aimed at treating serious or life-threatening diseases have shown preliminary clinical evidence that the drug has the potential to address unmet medical needs for the disease. Investigational medicines with RMAT are provided intensive interactions with the FDA during the product candidate's development process in addition to being eligible for rolling submission and priority review of the marketing application. 'It's gratifying to see another important benchmark reached in our work to combat pediatric brain cancer,' said Dr. Jeff Sperring, Chief Executive Officer at Seattle Children's. 'Our research is the foundation of progress to bring potential therapies to kids as fast as we can – and we're excited about the possibilities afforded by this designation.' BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024. About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis. 1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer. The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T-cells. About BrainChild Bio BrainChild Bio, Inc., is a kids-first, clinical-stage biotechnology company harnessing the power of CAR T-cell technology to treat tumors in the central nervous system, prioritizing pediatric indications with plans to expand into adult CNS tumors, specifically Glioblastoma and brain metastasis. BrainChild Bio was launched out of Seattle Children's Therapeutics program and founded on the work of Dr. Michael Jensen, a pioneer in the cancer immunotherapy field and previous Chief Therapeutics Officer at Seattle Children's. BrainChild Bio is advancing a next-generation CAR T-cell therapy platform for tumors of the CNS that weaves together synthetic technologies, including multiplex targeting and enhanced potency controls, to enable multiple targets in a single CAR T-cell therapy, novel transgenes to increase potency, delivery technology for durable efficacy, and streamlined CAR T-cell design and manufacturing. BrainChild Bio's lead drug candidate is BCB-276, an autologous CAR T-cell therapy that targets the immune checkpoint B7-H3, that is advancing in clinical trials for the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. BrainChild Bio's cell therapy approach using autologous B7-H3 CAR T-cell therapy has received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration (FDA). More information is available at _____________________________________________________________ 1 DIPG Registry.
Yahoo
29-04-2025
- Health
- Yahoo
Virginia Mason Franciscan Health and Seattle Children's expand pediatric, neonatal partnership
Virginia Mason Franciscan Health (VMFH) and Seattle Children's announced Tuesday they have expanded their longstanding partnership to enhance pediatric and mother-baby health care services across the Puget Sound region. The new strategic affiliation builds on nearly 15 years of collaboration between the two organizations and aims to improve access to expert physicians, specialized care teams, and seamless coordination for families, according to a joint statement. Access to high-quality health care remains a significant challenge in the Puget Sound region, VMFH and Seattle Children's noted. By joining resources and leveraging their combined network of care sites, both groups say they hope to bring advanced medical services closer to home for more patients. 'This affiliation marks a significant step forward in our mission to improve health care access for mothers, babies and children,' said Ketul J. Patel, CEO of Virginia Mason Franciscan Health and President of CommonSpirit Health's Northwest Region. 'Seattle Children's is a globally recognized leader in pediatric medicine, and we're honored to work alongside them to further elevate the standard of care in our communities.' Seattle Children's consistently ranks among the top ten children's hospitals in the United States, while VMFH hospitals are also nationally recognized for quality care. Their partnership has already brought neonatologists to VMFH Family Birth Centers, offering 24/7 onsite and remote support to care for the region's most vulnerable newborns without the need to transfer them to different facilities. The collaboration has also connected patients at VMFH's St. Joseph Medical Center in Tacoma to Seattle Children's pediatric cardiologists and infection prevention teams, allowing for on-site assessments of complex maternal and newborn conditions. Jeff Sperring, MD, Chief Executive Officer of Seattle Children's, said the expanded affiliation aligns with the hospital's broader mission. 'We are proud of our long-standing reputation for excellence in pediatric and neonatal care,' Sperring said. 'Virginia Mason Franciscan Health is a recognized leader in quality care and clinical excellence, and we're excited to work together to continue fulfilling our vision of helping every child live their healthiest and most fulfilling life possible.' As part of the expanded affiliation, perinatal, neonatal, and specialty services will grow across VMFH Birth Centers located in King, Pierce, and Kitsap counties. The goal is to support thousands of families while minimizing the separation of mothers and their newborns after delivery. The partnership will also focus on improving access to pediatric specialists across VMFH's nearly 300 care sites, strengthening connections between primary, specialty, ambulatory, and urgent care services. Officials said this effort will ensure children can get the right care at the right time, with seamless referrals to Seattle Children's specialists when needed.
Business Wire
22-04-2025
- Business
- Business Wire
FDA Grants Breakthrough Therapy Designation for BrainChild Bio's B7-H3 CAR T-Cell Therapy for Incurable Pediatric Brain Tumors
SEATTLE & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), today announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Breakthrough Therapy designation (BTD) by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This FDA designation was based on the promising overall survival benefit in patients with brain tumors treated with an autologous B7-H3 CAR T-cell therapy observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio's academic partner, Seattle Children's, and recently published in Nature Medicine. 'Breakthrough Therapy designation gives us the possibility to accelerate the development path for BCB-276 as a CAR T-cell therapy that can potentially transform the treatment of DIPG,' stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. 'This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating CNS brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases.' FDA grants Breakthrough Therapy designation to investigational medicines that demonstrate the potential to treat a serious or life-threatening condition and show preliminary clinical evidence that the drug may show substantial clinical improvement over available therapies. Investigational medicines with BTD are provided early and more frequent interactions with the FDA to discuss the product candidate's development plan in addition to being eligible for rolling submission and priority review of the marketing application. 'This designation is an important milestone for Seattle Children's and demonstrates our continued momentum in pediatric brain cancer research,' said Dr. Jeff Sperring, Chief Executive Officer of Seattle Children's. 'We harness the power of research to bring potential cures to kids faster, and we're excited by the early promise shown by our work with BrainChild Bio to advance a potential CAR T therapy.' BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024. About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis. 1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer. The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T cells. About BrainChild Bio BrainChild Bio, Inc., is a kids-first, clinical-stage biotechnology company harnessing the power of CAR T-cell technology to treat tumors in the central nervous system, prioritizing pediatric indications with plans to expand into adult CNS tumors, specifically Glioblastoma and brain metastasis. BrainChild Bio was launched out of Seattle Children's Therapeutics program and founded on the work of Dr. Michael Jensen, a pioneer in the cancer immunotherapy field and previous Chief Therapeutics Officer at Seattle Children's. BrainChild Bio is advancing a next-generation CAR T-cell therapy platform for tumors of the CNS that weaves together synthetic technologies, including multiplex targeting and enhanced potency controls, to enable multiple targets in a single CAR T-cell therapy, novel transgenes to increase potency, delivery technology for durable efficacy, and streamlined CAR T-cell design and manufacturing. BrainChild Bio's lead drug candidate is BCB-276, an autologous CAR T-cell therapy that targets the immune checkpoint B7-H3, that is advancing in clinical trials for the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. More information is available at
