Baylor College of Medicine

HOUSTON, Oct. 30, 2024 /PRNewswire/ -- Prominently featured in The Inner Circle, Steven Lorch is acknowledged as a Pinnacle Life Member for his contributions to the field of Pediatric Cardiology. Dr. Lorch attended Emory University in Atlanta where he received a Bachelor of Science degree. He earned a medical degree from Emory University, School of Medicine; completed a residency in pediatrics at Baylor College of Medicine/Texas Children's Hospital; a fellowship in pediatric cardiology at Washington University/St. Louis Children's Hospital. Board-certified in pediatric cardiology, Dr. Lorch explained that as a pediatric cardiologist, he sees infants, young children, and adolescents on an outpatient basis. He treats children for cardiac abnormalities, heart murmurs, tachycardia, and palpitations and works closely with the families and parents of these children, educating them on their conditions. A proven professional, Dr. Lorch specializes in caring for fetuses, infants, children and adolescents with cardiovascular or cardiac abnormalities. He is experienced in both inpatient and outpatient settings and explained that the scope of conditions cared for by pediatric cardiologists is large and includes congenital heart defects; heart muscle disorders; rhythm disturbances; and hypertension. He said that pediatric cardiology is a medical specialty that involves the evaluation, diagnosis, and treatment of congenital (present at birth) and acquired cardiovascular (heart and blood vessel) problems of the fetus, infant, child, adolescent, and young adults. Pediatric cardiologists, like Dr. Lorch, diagnose and treat children with heart conditions before they are born; through childhood; and into adulthood. His clinical interests include pediatric and congenital heart disease, fetal echocardiography, and preventive cardiology. Dedicated to professional networking and staying abreast of the latest innovations in his field, Dr. Lorch is affiliated with Woman's Hospital of Texas; Memorial Hermann Memorial City Medical Center; Memorial Hermann Katy Hospital; Memorial Hermann Northeast Hospital; Memorial Hermann Sugar Land Hospital; and St. Joseph Health College Station Hospital. Prior to his post at Pediatrix Cardiology of Houston, Dr. Lorch served as assistant head of the Pediatric Echocardiography Laboratory at the University of Texas Health Science Center in Houston. He is a Diplomate of the American Board of Pediatrics (ABP) and explained that the mission of the ABP is to advance child health by certifying pediatricians who meet standards of excellence and are committed to continuous learning and improvement. The doctor is a member of the American Society of Echocardiography; American College of Cardiology; Harris County Medical Society; Texas Medical Association. When considering his success, Dr. Lorch notes that his mentors include Achiau Ludomirsky, MD who was his mentor and division director in St. Louis. Aside from his professional pursuits, Dr. Lorch is a parent volunteer with the Boy Scouts of America Troop 55 in Houston. He has been married to Mrs. Clare Gentry for 22 years and they have one son and daughter. For more information about Dr. Lorch's practice, please visit his website at . Contact: Katherine Green, 516-825-5634, [email protected] SOURCE The Inner Circle WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Wholly owned first-in-class program anticipated to enter clinical trials in first half of 2025 THOUSAND OAKS, Calif., Oct. 30, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Capsida for CAP-002. CAP-002 is the company's lead investigational gene therapy for the treatment of developmental and epileptic encephalopathy (DEE) due to syntaxin-binding protein 1 (STXBP1) mutations. CAP-002 is a novel, first-in-class IV-administered gene therapy using one of Capsida's proprietary engineered capsids designed to achieve brain-wide neuronal expression of the STXBP1 protein while significantly detargeting the liver. CAP-002 is currently in IND-enabling studies. "FDA's granting Orphan Drug Designation signals the significant potential that CAP-002 demonstrates based upon our preclinical data," said Peter Anastasiou, Chief Executive Officer of Capsida Biotherapeutics. "There are no disease-modifying therapies available for STXBP1 developmental and epileptic encephalopathy and CAP-002 could be the first, bringing hope for patients and families affected by this devastating disorder." The STXBP1 protein is present in every neuron in the brain and is essential for normal neurotransmission. A mutation in the STXBP1 gene is associated with treatment-resistant seizures, severe developmental delay and intellectual disability, motor abnormalities, and sudden unexpected death in epilepsy (SUDEP). Preclinical pharmacology studies have established the potential to treat and even fully correct the disease through gene supplementation. These studies demonstrated rescue of these neurological phenotypes was dependent on supplementation of STXBP1 in neurons throughout the brain at levels not achievable by wild-type serotypes, such as AAV9. Capsida conducted these studies with a proprietary murine disease model in collaboration with Mingshan Xue, Ph.D., associate professor of neuroscience and molecular and human genetics at Baylor College of Medicine, as well as a member of the Cain Foundation Laboratories and the Duncan Neurological Research Institute at Texas Children's Hospital. "Receiving Orphan Drug Designation moves us one step closer to our goal of transforming the lives of people living with STXBP1 developmental and epileptic encephalopathy," said Swati Tole, M.D., Chief Medical Officer at Capsida. "We are committed to advancing CAP-002, with our IND filing in the first half of 2025, and bringing novel gene therapies to people with high unmet medical needs." The FDA's Orphan Drug Designation is intended to encourage the development of treatments for rare diseases, defined as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation may shorten the clinical development path through closer FDA collaboration and potential qualification for expedited development programs. This designation also provides the company with certain benefits, including tax credits for qualified clinical trials, exemption from user fees, and potential seven-year market exclusivity upon FDA approval. About STXBP1 Developmental and Epileptic Encephalopathy Developmental and epileptic encephalopathy caused by STXBP1 mutations is estimated to affect up to one in 26,000 children globally. It is associated with treatment resistant seizures, severe developmental delay and intellectual disability, motor abnormalities, and sudden unexpected death in epilepsy (SUDEP). There are no approved treatments. About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including a potential -first-in-class treatment for STXBP1 developmental and epileptic encephalopathy and a best-in-class treatment Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at . SOURCE Capsida Biotherapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Mission BioCapital and 4BIO Capital led the financing with significant participation from new and existing investors, raising over $51 million to date Funds will support Phase 2 development of March Bio’s lead CD5 CAR-T therapy, MB-105, for T-cell lymphoma and pipeline candidates HOUSTON, Oct. 23, 2024 (GLOBE NEWSWIRE) -- March Biosciences (March Bio), an emerging clinical stage biotechnology company committed to combating challenging cancers and other diseases, today announced the close of a $28.4 million oversubscribed Series A financing. Mission BioCapital and 4BIO Capital led the financing, with participation from KdT Ventures, Alexandria Venture Investments, Volnay Therapeutics, Modi Ventures, and Mansueto Investments. Existing investors TMC Venture Fund, Cancer Focus Fund, and Small Ventures also participated in the round. March Bio has raised over $51 million to date inclusive of all dilutive and non-dilutive funding. Since its inception as a spinout of the Center for Cell and Gene Therapy (Baylor College of Medicine, Houston Methodist Hospital, Texas Children’s Hospital), March Bio has rapidly advanced its innovative autologous chimeric antigen receptor T-cell (CAR-T) therapy, MB-105, in development for the treatment of relapsed and refractory CD5 positive T-cell lymphoma. MB-105 is specifically engineered to overcome major hurdles related to T-cell targeting by overcoming T-cell fratricide while maintaining high potency against CD5 positive tumor cells. MB-105 has demonstrated a favorable safety profile and durable remissions in relapsed T-cell lymphoma patients in a Phase 1 clinical trial at Baylor College of Medicine, with plans to begin a Phase 2 clinical trial in early 2025. Proceeds from the financing will support the Phase 2 clinical development of MB-105 to expand on this data with optimized manufacturing processes. “This oversubscribed financing enables us to advance our first-in-class CAR-T therapy, MB-105, into a Phase 2 trial for T-cell lymphoma – an indication with an exceptionally poor prognosis and few treatment options,” said Sarah Hein, Co-Founder and Chief Executive Officer of March Biosciences. “With the support and confidence of our investors, we are not only advancing our lead program but also expanding our pipeline, underscoring our commitment to delivering best-in-class therapies to patients that can change the treatment paradigm for these challenging cancers.” The new financing coincides with an announcement that the company has partnered with Volnay Therapeutics, a cell therapy venture studio led by a team of highly experienced cell therapy development veterans. March Biosciences will work with Volnay for technical oversight to develop a robust and scalable manufacturing process for MB-105 through clinical development and potential commercialization, a critical component of a successful autologous cell therapy program. Concurrent with the partnership announcement, Volnay Co-Founder and CEO Stefan Wildt, who held key R&D leadership positions in cell and gene therapy units at Novartis and Takeda, is joining March Bio’s Board of Directors. “For far too long, T-cell cancers have been an innovation desert with patients facing a dismal prognosis. March Bio’s innovative autologous CAR-T approach brings patients new hope,” said Owen Smith, Partner at 4BIO Capital. “MB-105 is specifically engineered for relapsed and refractory CD5 positive T-cell lymphomas and I am delighted that this targeted approach combined with the incredible team led by Sarah is moving rapidly into Phase 2 to bring this exciting new treatment to patients. We are honored to be a co-lead investor in March Bio and to help support the company as it continues in its mission to bring transformative therapies to those in urgent need.” “The team at March Biosciences is leveraging powerful science and promising clinical data to tackle cancers with significant unmet need,” said Cassidy Blundell, Partner at Mission BioCapital. “We're excited to support their journey and believe their focused approach with MB-105 could lead to significant breakthroughs in the CAR-T space.” Cassidy Blundell of Mission BioCapital and Owen Smith of 4BIO Capital are also joining March Bio’s Board of Directors. The financing will also provide resources for the ongoing development of undisclosed pipeline products, as well as for general corporate proceeds. About March BiosciencesHouston-based March Biosciences, launched from the Center for Cell and Gene Therapy (Baylor College of Medicine, Houston Methodist Hospital, Texas Children’s Hospital), is dedicated to addressing challenging cancers unresponsive to current immunotherapies. Its lead asset, MB-105, is a CD5-targeted CAR-T cell therapy currently in Phase 1 trials in patients with refractory T-cell lymphoma and leukemia, with promising signals of efficacy and safety to date. A Phase 2 trial is expected to begin next early year. The company has raised over $50M to date, inclusive of this current financing and support from the Cancer Prevention & Research Institute of Texas (CPRIT) and the NIH SBIR program. Learn more at www.march.bio. About 4BIO Capital4BIO Capital (“4BIO”) is an international venture capital firm focused on investing in advanced therapies, including genomic medicines and other emerging technologies, to unlock the treatments of the future. 4BIO’s objective is to invest in, support, and grow early-stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome. The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals including Nature, The Lancet, Cell, and the New England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space. For more information, visit www.4biocapital.com. About Mission BioCapitalMission BioCapital is a biotech venture capital firm focused on making pivotal early-stage investments in life sciences companies. They support portfolio companies through a unique combination of shared lab space, capital investment, and access to strategic partners. With offices in the nation’s leading life sciences clusters in Cambridge, MA and San Francisco, Mission BioCapital is dedicated to helping entrepreneurial scientists build successful companies, from idea to exit. Learn more at missionbiocapital.com. About Volnay TherapeuticsVolnay Therapeutics is a biotech venture builder dedicated to accelerating the development of innovative cell and gene therapies. With decades of experience in drug development, Volnay identifies promising therapeutic assets and enabling technologies with disruptive potential and provides both capital and operational expertise to drive these ventures forward. By focusing on innovation in product manufacturing, commercial viability and capital efficiency, Volnay aims to deliver significant value to patients and investors alike. Visit www.volnaytx.com. Forward-Looking StatementThis press release contains forward-looking statements that involve risks and uncertainties, including those related to the clinical development of MB-105 and other potential therapies. Actual results may differ materially from those projected in these forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, the progress of clinical trials, regulatory approvals, market demand for new therapies, and competitive dynamics in the biotechnology sector. Contacts: Corporate:Sarah HeinChief Executive Officer sarah@march.bio Media:Jon YuMarchBioPR@icrhealthcare.com