Developmental Disabilities

The innovative food-as-medicine platform will help support eligible North Carolinians with serious mental health needs transitioning from institutional care settings to independent living. STAMFORD, Conn., March 19, 2024 /PRNewswire/ -- Alliance Health, a North Carolina-based behavioral health care services managed care company, announced today that it has entered into a strategic partnership with NourishedRx, a digital health and nutrition company based in Stamford, Connecticut, to support eligible members enrolled in North Carolina's Transitions to Community Living (TCL) program. The new partnership will enable Alliance Health to better serve TCL program members transitioning from institutional care settings into community living by providing them with nutritious home-delivered food and associated nutritional support services. Continue Reading Alliance Health "Access to quality food and nutrition is an indispensable component of well-being and has a measurable impact on physical and mental health, including loneliness and depression," said Lauren Driscoll, NourishedRx Founder and CEO. "When combined with coaching, education, and social support, such services catalyze confidence in self-care and self-efficacy that is foundational to successful independent living." The TCL program supports eligible North Carolina adults living with serious mental illness who chose to live outside institutional care settings. Eligible members may have mental health disorders, substance abuse disorders, intellectual/developmental disabilities, or traumatic brain injuries. The TCL program combines affordable housing with intensive, coordinated services, including case management, peer support, and community integration support, to help program participants live and thrive in their communities. Currently, Alliance Health supports over 1,000 households through the TCL program. The partnership's goals are to positively impact tenancy and independent living, improve participants' nutrition security, support health equity, and improve self-reported health outcomes. "In NourishedRx, we've identified a partner with a proven track record of working successfully with vulnerable populations at scale, and a team and platform that can deliver the results we want to see in this innovative program," said Ann Oshel, SVP of Community Health and Well Being, of Alliance Health. "We're excited to engage with NourishedRx in our effort to improve the health and lives of North Carolinians in need of these services." About Alliance Health Alliance Health is the managed care organization for publicly funded behavioral health care services for the people of Cumberland, Durham, Johnston, Harnett, Orange, and Wake counties in central North Carolina as well as Mecklenburg County. Currently, Alliance works with a network of over 4,000 private providers to serve the needs of almost 190,000 Medicaid-eligible and state-funded individuals among a population of over 3.5M. On July 1, 2024, Alliance will begin operating as a Tailored Plan as part of North Carolina's Medicaid transformation. In that role, Alliance will manage all the health care needs – physical, behavioral, and pharmaceutical – for individuals with severe mental illnesses, substance use disorders, or long-term care needs, including intellectual/developmental disability and traumatic brain injury. About NourishedRx NourishedRx is on a mission to eradicate poor diet and nutrition insecurity as top drivers of death, disease, and disparities. Founded in 2019, NourishedRx is a digital health and nutrition company that helps people live healthier lives and supports health equity. Leveraging the connective power of food, NourishedRx partners with healthcare organizations to nourish their most vulnerable members, build health relationships, and support lasting dietary behavior change. Visit Media Contact: Cathy Giandurco [email protected] SOURCE NourishedRx

Designation grants access to enter the Innovative Licensing and Access Pathway (ILAP) Five patients treated with FBX-101 show positive safety and efficacy results, including significant improvements in motor function Forge’s CMO, Dr. Maria Escolar, to present updated FBX-101 clinical data during the Advanced Therapies 2024 conference in London March 19-20 Designation provides opportunities for further global acceptance of Forge’s manufacturing process and facility through additional reviews and inspections COLUMBUS, Ohio--(BUSINESS WIRE)-- Forge Biologics, a member of Ajinomoto Bio-Pharma Services and a leading manufacturer of genetic medicines, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovation Passport designation to the Company’s novel AAV gene therapy program, FBX-101, to enter the Innovative Licensing and Access Pathway (ILAP). FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two. The ILAP is intended to accelerate regulatory and market access interactions in the UK. Maria Escolar, M.D., Forge’s Chief Medical Officer, will discuss the significance of achieving this regulatory designation and present a poster on updated data from REKLAIM, an early phase clinical trial for FBX-101, during the Advanced Therapies 2024 conference being held in London, March 19-20, 2024. “Considering the rapid progression of Krabbe disease in young patients and the positive results we’ve observed in the REKLAIM trial, we are pleased to receive the Innovation Passport designation which will enable Forge to further accelerate the clinical development of FBX-101,” stated Dr. Escolar. “ILAP was created to provide access to tools to decrease the time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions.” The REKLAIM clinical trial is evaluating the safety and efficacy of FBX-101 in patients with infantile and late infantile Krabbe disease. Children assessed in REKLAIM have received intravenous FBX-101 in addition to the current standard of care, hematopoietic stem cell transplantation (HSCT). Clinical data demonstrate that FBX 101 has been well tolerated and has an excellent safety pro the treatment of five patients. Results show improvements in motor function in all patients, the most meaningful clinical outcome for this disease. FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease. In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation. ILAP is intended to accelerate regulatory and market access interactions in the UK for patient populations with significant need, leading to accelerated patient access to novel therapies. It also provides access to a range of UK development services and tools, including frequent MHRA interactions, accelerated Marketing Authorization Application (MAA) assessment, a Target Development Profile, expanded licensing routes, and more, creating a roadmap for advancement of therapies through regulatory approval. Regulatory review by the MHRA through ILAP provides opportunities for further global acceptance of Forge’s manufacturing process and facility though additional reviews and inspections, including Compliance Readiness Inspections that provide custom and pragmatic supervisory and licensing inspections specific to the GMP guidelines. Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK. This access to global health authority resources will enable an added level of assurance to clients utilizing Forge’s CDMO services with integrated regulatory support. About Krabbe Disease Krabbe disease is a rare neurodegenerative disease affecting about 1-2.5 in 100,000 people in the US. Krabbe disease is caused by autosomal recessive mutations in the galactocerebrosidase (GALC) gene, an enzyme responsible for the breakdown of certain types of sphingolipids, such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells, specifically in cells insulating the nerves in the brain and peripheral nervous system, causing rapid demyelination. Krabbe disease initially manifests in young patients as irritability, developmental delay, and progressive muscle weakness. Symptoms rapidly advance to difficulty swallowing, breathing, and regression of neurodevelopment followed by seizures, vision and hearing loss. Infantile Krabbe disease (0-12 months of age at onset) usually leads to death in untreated patients by two years of age. Late Infantile patients (12-36 months of age at onset) usually die by the age of six. The current standard of care, hematopoietic stem cell transplantation (HSCT), has been shown to stabilize cognitive decline and significantly improve long-term neurological outcomes. However, HSCT does not correct the peripheral neuropathy that is progressive as the patient grows, leading to loss of gross motor skills and eventually death. About FBX-101 FBX-101 was developed to treat children with Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is delivered intravenously after HSCT infusion. The vector delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system and has shown to functionally correct the central and peripheral neuropathy, improve myelination and gross motor function, and significantly prolong lifespan in animal models. This approach also has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies and extend the duration of gene transfer. About the REKLAIM Trial REKLAIM is a nonblinded, non-randomized, early phase dose escalation clinical trial currently enrolling children with asymptomatic infantile and symptomatic late infantile Krabbe disease to investigate the safety and efficacy of a single intravenous infusion of FBX-101 administered more than 21 days after HSCT, the current standard of care. Data from extensive natural history subjects will be used to compare as the control group. More information on the REKLAIM trial can be found online at . About Forge Biologics Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 200,000L of manufacturing capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit .

SOLANA BEACH, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine, today congratulates its partner PTC Therapeutics, Inc. on completion of its BLA submission to the U.S. Food and Drug Administration (FDA) for the approval of Upstaza™ (eladocagene exuparvovec), an investigational treatment for AADC Deficiency. If approved, Upstaza™ would become the first therapy to treat AADC Deficiency in the United States. “AADC Deficiency is a devastating rare pediatric movement disorder that causes significant developmental delays and autonomic symptoms starting from birth. Patients with AADC deficiency are at a high risk of death in the first decade of life,” stated Jeremy Stigall, Chief Business Officer at ClearPoint Neuro. “The Upstaza™ BLA is the first filing for FDA approval of a treatment that addresses this devastating condition. Through our partnership with PTC, we are demonstrating our commitment to drive progress for the AADC Deficiency community.” About aromatic L-amino acid decarboxylase (AADC) deficiency AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life – physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping. The lives of affected children are severely impacted and shortened. Ongoing physical, occupational and speech therapy, and interventions, including surgery, also are often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems. About the SmartFlow® Cannula With over 7,000 cannulas sold to date, SmartFlow is the only co-labeled device to gain approval by a regulatory agency for delivery of an approved gene therapy to the brain. The industry-leading cannula is used by many of ClearPoint Neuro’s 50+ pharmaceutical, academic, and biotech partners to bypass the blood brain barrier and deliver therapeutics to regions of interest using Convection Enhanced Delivery (CED) under direct image guidance. The SmartFlow Cannula has 510(k) clearance from the FDA for use in the United States for the aspiration of cerebrospinal fluid or injection of the chemotherapy drug Cytarabine into the ventricles. It has also been CE marked to deliver approved fluids into the brain and for aspiration of cerebrospinal fluid, and holds regulatory clearance for clinical use in Israel and Brazil. SmartFlow is utilized in approved clinical and preclinical studies for various research and drug trials. About ClearPoint Neuro ClearPoint Neuro is a device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine. The Company uniquely provides both established clinical products as well as pre-clinical development services for controlled drug and device delivery. The Company’s flagship product, the ClearPoint Neuro Navigation System, has FDA clearance and is CE-marked. ClearPoint Neuro is engaged with healthcare and research centers in North America, Europe, Asia, and South America. The Company is also partnered with the most innovative pharmaceutical/biotech companies, academic centers, and contract research organizations, providing solutions for direct CNS delivery of therapeutics in pre-clinical studies and clinical trials worldwide. To date, thousands of procedures have been performed and supported by the Company’s field-based clinical specialist team, which offers support and services to our customers and partners worldwide. For more information, please visit Forward-Looking Statements This press release contains forward-looking statements within the context of the federal securities laws, which may include the Company’s expectation for the future market of its products and services, and other performance and results. These forward-looking statements are based on management’s current expectations and are subject to the risks inherent in the business, which may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to: global and political instability, supply chain disruptions, labor shortages, and macroeconomic and inflationary conditions; future revenue from sales of the Company’s products and services; the Company’s ability to market, commercialize and achieve broader market acceptance for new products and services offered by the Company; the ability of our biologics and drug delivery partners to achieve commercial success, including their use of the Company’s products and services in their delivery of therapies; the Company’s expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; the Company’s ability to obtain additional funding to support its research and development programs; the ability of the Company to manage the growth of its business; the Company’s ability to attract and retain its key employees; and risks inherent in the research, development, and regulatory approval of new products. More detailed information on these and additional factors that could affect the Company’s actual results are described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, which has been filed with the Securities and Exchange Commission. The Company does not assume any obligation to update these forward-looking statements. A photo accompanying this announcement is available at Contact: Media Contact: Jacqueline Keller, Vice President of Marketing (888) 287-9109 ext. 4 info@clearpointneuro.com Investor Relations: Danilo D’Alessandro, Chief Financial Officer (888) 287-9109 ext. 3 ir@clearpointneuro.com ClearPoint Neuro ClearPoint Neuro Navigation Platform for Drug Delivery