Genetic Diseases, Inborn

MB-111 is a potential first-in-class in vivo ultracompact CRISPR therapy for Familial Chylomicronemia Syndrome and Severe Hypertriglyceridemia Dr. Brown brings deep expertise in nucleic acid drug development as Mammoth advances its lead program, MB-111, to IND-enabling stage and continues to build a leading company in genetic medicines BRISBANE, CA, USA I May 05, 2025 I Mammoth Biosciences, Inc. , a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced the nomination of its first clinical development candidate, MB-111, and the appointment of biotechnology industry veteran, Bob D. Brown, Ph.D., to its Board of Directors. MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. Share MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. MB-111 has the potential to be a first-in-class, one-time treatment for patients with very high triglycerides including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). MB-111 is designed to permanently disrupt the expression of the APOC3 gene in the liver, and thus to reduce expression of ApoC-III protein. ApoC-III is a critical driver of lipid metabolism and disrupting its production has been shown to reduce plasma triglycerides in patients with pathologically elevated levels, including those with FCS and SHTG. These patients suffer from recurrent episodes of acute pancreatitis, leading to frequent hospitalizations, as well as an increased risk of cardiovascular disease. Mammoth Biosciences is on track to initiate IND-enabling studies this year. “The nomination of our lead program as a development candidate is a major milestone for Mammoth and the gene editing field, as well as the first proof point of the therapeutic potential of our novel ultracompact CRISPR systems and gene editing technologies,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences. “The preclinical data on MB-111 is compelling, and we’re excited about its potential to be a first-in-class genetic cure for debilitating diseases such as FCS and SHTG. We believe Dr. Brown’s extensive drug discovery and development experience, and track record of building leading companies in siRNA and antisense oligonucleotide therapeutics across rare and chronic diseases will be invaluable to our goal of increasing the number of patients who can derive benefit from Mammoth Biosciences’ genetic medicines.” Dr. Bob D. Brown brings over 30 years of experience in multidisciplinary biotechnology research and drug development, including in the US and ex-US jurisdictions. Most recently, Dr. Brown served as Chief Scientific Officer and Executive Vice President of R&D at Dicerna Pharmaceuticals, an RNAi-focused therapeutics company that was acquired by Novo Nordisk. At Novo, he served as President and Head of the Dicerna Transformation Research Unit and SVP. During his time at Dicerna, he led the discovery and early clinical development of numerous genetic medicines, including nedosiran for the treatment of primary hyperoxaluria, RG6346 for the treatment of chronic hepatitis B, belcesiran for the treatment of alpha-1 antitrypsin deficiency, and several other drug candidates now in the clinical development pipelines of large pharmaceutical companies. Prior to Dicerna, Dr. Brown held various positions at Genta, a clinical-stage antisense oligonucleotide therapeutics company, most recently as its Vice President of Research and Technology. Previously, he was a co-founder and Vice President of R&D of Oasis Biosciences, which was acquired by Gen-Probe. Dr. Brown is an inventor or co-inventor of more than 85 issued US patents, and earned a Ph.D. in molecular biology from the University of California, Berkeley and B.S. degrees in chemistry and biology from the University of Washington, Seattle. “I’m honored to join Mammoth Biosciences at such a pivotal moment, as CRISPR technology stands ready to transform healthcare and redefine the future of medicine,” said Dr. Brown. “I look forward to working with the exceptional Mammoth Biosciences team to harness the power of their groundbreaking platform to tackle some of the most pressing medical challenges and improve patients’ lives. Drawing on my experiences in drug discovery and clinical development, I aim to help advance Mammoth Biosciences’ ultracompact CRISPR gene editing therapies—starting with MB-111—toward clinical application.” About Mammoth Biosciences Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact systems are designed to be more specific and enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, reverse transcriptase editing, and epigenetic editing. The company is building out its wholly owned pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology platform. Mammoth Biosciences’ deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the promise of CRISPR technologies. SOURCE: Mammoth Biosciences

SANTA BARBARA, Calif., May 5, 2025 /PRNewswire/ -- Cottage Health has partnered with Clearway Health, a specialty pharmacy services company, to develop a specialty pharmacy program that enhances patient access to medications with concierge-level service at a lower cost. This collaboration brings personalized pharmacy support to Cottage Health's specialty outpatient clinics, enhancing patient access to medications. Continue Reading Cottage Health partners with Clearway Health to develop a specialty pharmacy program, enhancing access to medications. Post this “Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications,” said Lisa Moore, executive vice president and chief operating officer at Cottage Health. “This ensures our patients receive the therapies they need while reducing barriers and improving outcomes.” The program is designed to simplify the prescription process by assisting with insurance approvals, financial assistance, medication delivery, and ongoing support, making it easier for patients to manage their treatment without delays or added stress. As part of the partnership, pharmacists and patient liaisons are integrated directly within Cottage Health's specialty clinics, working closely with patients to navigate insurance coverage, secure financial assistance and manage prior authorizations—alleviating administrative burdens for both patients and providers. "Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications," said Lisa Moore, executive vice president and chief operating officer at Cottage Health. "This ensures our patients receive the therapies they need while reducing barriers and improving outcomes." The program has already launched at the Cottage Cystic Fibrosis Clinic and pediatric clinics, where patient liaisons are assisting patients with accessing both specialty and non-specialty medications. Specialty medications typically require special handling, administration and monitoring and are used to treat chronic conditions such as multiple sclerosis, cystic fibrosis, cancer and inflammatory conditions. "Clearway Health is pleased to work alongside Cottage Health to provide their patients and community with transformative specialty pharmacy services," said Allison Arant, senior vice president, client development and marketing, Clearway Health. "Cottage Health's patient-first approach and commitment to helping patients get back to living their lives directly coincides with our ability to improve access to care and manage the complex medical needs of patients. Every patient deserves the care we provide. We celebrate and congratulate Cottage Health for choosing to build this program and offer specialty pharmacy services to their patients." Future plans include expanding specialty pharmacy services to other Cottage Health clinics and opening an outpatient retail and specialty pharmacy, further enhancing medication access and convenience for patients. About Clearway Health Clearway Health's specialty pharmacy services create sustainable assets and build lasting legacies for hospitals and health systems, providing a service that every patient deserves. Our team began at Boston Medical Center Health System, improving access to care and managing the complex medication needs of vulnerable patient populations. Equipped with this unique experience to solve challenges, Clearway Health partners with other hospitals and health systems to provide transformative specialty pharmacy services that put patients first. Our strategic partnerships build and strengthen the infrastructure for specialty pharmacy programs through improving operational workflows, implementing clinical programs, hiring and embedding skilled pharmacists and patient liaisons into the clinical team, providing proprietary software technology, unlocking drug access and leading accreditation processes. Clearway Health delivers a clinically driven service that eliminates barriers to medication access, improves the health of patients and communities, grows revenue at healthy margins for health systems and reduces costs for patients. To learn more, visit . About Cottage Health | CottageHealth.org Cottage Health is a not-for-profit health system providing advanced medical care on California's Central Coast. In the past year, its hospitals in Goleta, Santa Barbara and Santa Ynez Valley cared for 20,733 inpatient admissions, 91,649 emergency department visits and 2,005 newborn deliveries. With more than 700 physicians, including specialists typically found only at university medical centers, Cottage offers expert, comprehensive care. As a teaching institution, it trains physicians through residency programs in medical, surgical, pediatric and radiology specialties. Cottage Health is home to the Central Coast's only Level I adult and Level II pediatric trauma center and an advanced comprehensive stroke center. Its specialty services include Cottage Children's Medical Center, Cottage Rehabilitation Hospital, the Santa Barbara Neuroscience Institute, Cottage Heart & Vascular Center and Cottage Center for Orthopedics. Beyond its hospitals, Cottage expands care through primary and specialty clinics, urgent care centers and 24/7 provider access via Cottage Virtual Care. SOURCE Clearway Health WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study EAP Supports Real-world Data Collection Concurrent to Ongoing Clinical Studies of SPG302 LOS ANGELES, May 5, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that it has received notification from the Food and Drug Administration (FDA) to begin its Expanded Access Program (EAP) for SPG302 in the United States (U.S.) for people living with Amyotrophic Lateral Sclerosis (ALS) that meet program eligibility criteria. SPG302 is being developed as the first synaptic regenerative approach to treating ALS, with the potential to slow or reverse declines in cognitive and motor function. Spinogenix CEO and Founder Dr. Stella Sarraf stated, "FDA clearance to launch this new EAP supports its awareness of SPG302's safety and potential efficacy, and the need for ALS patients to have access to novel treatments. This EAP reflects our dedication to giving hope to people living with ALS and their families, providing treatment to those who do not qualify for, or may be unable to participate in, our ongoing clinical trial. Innovation is urgently needed to tackle ALS, a disease with devastating impact, and we are committed to ensuring that no patients who may benefit are left behind." EAPs are designed to allow individuals with serious or life-threatening conditions to access investigational treatments outside of a clinical trial that are not yet approved by the FDA. Dan Doctoroff, founder and chairman of Target ALS, and the first individual to receive SPG302 in the U.S. as part of a separate EAP protocol commented, "The notion that ALS is an untreatable disease is outdated. My own experience with SPG302, the first synaptic regenerative therapy in clinical development, has shown that partial stabilization of the disease is a possibility. I am thrilled that the FDA has recognized the value of allowing Spinogenix to expand access to SPG302, allowing others battling ALS to have a similar beneficial experience." In addition to serving the ALS community by providing access to an innovative investigational therapy, the EAP enables the collection of real-world data on SPG302 safety and efficacy that can support its clinical development. The FDA previously granted SPG302 Orphan Drug Designation for the treatment of ALS. Spinogenix recently completed a Phase 2 ALS trial in Australia, and patients have been offered a continuation of treatment in an open label extension. Additional information on the trial (NCT05882695) and open label extension (NCT06903286) may be found on ClinicalTrials.gov. About SPG302 SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function. SPG302 has been granted U.S. FDA Orphan Drug Designation for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense. Spinogenix has completed a Phase 1/2 study in Australia (NCT05882695) assessing the safety, pharmacokinetics and pharmacodynamics of once-daily dosing of SPG302 in healthy volunteers and up to 48 weeks of treatment in ALS patients. Additional information on the global clinical trials evaluating SPG302 for the treatment of schizophrenia and Alzheimer's disease can be found on ClinicalTrials.gov (NCT06442462 and NCT06427668). About Expanded Access to SPG302 Access to SPG302 is available in the U.S. for certain adults with ALS who meet eligibility criteria for participation in the FDA-authorized Expanded Access Program for SPG302. More information about the U.S. EAP can be requested through [email protected]. About Spinogenix Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope. Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug designation for both ALS and FXS, as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at or follow us on LinkedIn. Media Relations Nechama Rosengarten FINN Partners [email protected] Investor Relations Dan Albosta Spinogenix, Inc. [email protected] SOURCE Spinogenix WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED