Hereditary Central Nervous System Demyelinating Diseases

NATICK, Mass.--(BUSINESS WIRE)-- AffyImmune Therapeutics, Inc., a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies, announced today the appointment of Matt Britz to Chief Executive Officer and Pete Gelinas as Senior Vice President of Chemistry, Manufacturing, and Controls. This press release features multimedia. View the full release here: Matt Britz, CEO, AffyImmune (Photo: Business Wire) “Matt has been an instrumental part of AffyImmune’s growth in the past two years,” said Simone Song, Chairperson of the Board of Directors, AffyImmune, and Founder and Senior Partner at ORI Capital. “AffyImmune is a company incubated by ORI Capital. Matt has executed a focused business strategy supported by insights generated from our big data platform ORIzon, which has led to promising early clinical results for AffyImmune’s lead program AIC100 in advanced thyroid cancer. Matt is well-suited to lead AffyImmune as CEO as the Company accelerates clinical development of AIC100 in thyroid cancer, and in other indications with high unmet medical need. I am also thrilled to welcome Pete to our leadership team at a critical time as we scale up CMC efforts to support our ongoing trial and potential indication expansion. Pete’s extensive BLA experience will be invaluable as AffyImmune moves to the next stage of growth.” “I’m honored to lead AffyImmune’s efforts in advancing affinity-tuned CAR T therapies, building upon the solid foundation we’ve established in the last few years,” said Britz. “Together, we're well-positioned to make significant advancements in addressing the challenges of solid cancers to improve patient outcomes." Matt Britz joined AffyImmune in June 2021 as Senior Vice President (SVP), Business Development and was promoted to Chief Operating Officer in January 2022. He brings 28 years of demonstrated experience in life sciences, and was previously SVP and Head of Business Development at Minerva Biotechnologies, where he led teams across corporate development, manufacturing, clinical operations and regulatory affairs. Prior to Minerva Biotechnologies, Matt held scientific, consulting, and business development positions at Merck, Pfizer, Equinox Group, Health Advances, and Envoy Therapeutics, and played a crucial role in the acquisition of Envoy Therapeutics by Takeda. Matt has expertise leading teams in the initial stages of drug discovery all the way through to development and commercialization. Matt has an MBA from Washington University St. Louis, and a BS in Chemical Engineering and Biology from MIT. Pete Gelinas brings over 15 years of extensive experience in Manufacturing and CMC Product Development to AffyImmune. Previously, he served as the Head of Manufacturing and Technical Operations at ElevateBio, where he led technology transfer, CMC strategy, and oversight of routine clinical manufacturing. During his tenure at bluebird bio, he led both the MSAT and CMC teams for Skysona, a treatment for Cerebral Adrenoleukodystrophy (CALD). He has executed numerous late-phase global technology transfer deals and played a pivotal role in driving the drug product process validation efforts critical to the successful Marketing Authorization Application (MAA) for Zynteglo. Peter has also held various positions at the Connell O’Reilly Cell Manipulation Core Facility (CMCF) at Dana Farber Cancer Institute (DFCI), where he contributed to the development and manufacturing of several gene and cell-based therapies. “I am thrilled to join AffyImmune at this next stage of growth, with the opportunity to contribute my expertise in manufacturing to advance this pipeline for patients with solid cancers,” said Gelinas. “I look forward to making a difference in the lives of patients and their families, and to deliver our life-changing therapies in areas with high unmet medical need.” About AffyImmune Therapeutics AffyImmune is a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies to transform the lives of patients with cancer. The Company’s proprietary technology utilizing ICAM1/LFA-1 interaction can potentially address all critical CAR T challenges such as toxicity, antigen loss, T-cell trafficking and exhaustion, while also allowing in vivo tracking of the CAR T cells. Its lead candidate, AIC100, is a ICAM1-directed CAR T therapy being developed for patients with advanced thyroid cancer. AffyImmune is developing a focused pipeline of therapeutics targeting ICAM-1 in oncologic indications with high unmet medical need. AffyImmune is based in Natick, MA. For more information, visit .

Pictured: FDA signage at its headquarters in Maryland/iStock, hapabapa The FDA on Monday approved Orchard Therapeutics’ autologous gene therapy atidarsagene autotemcel, which will now be marketed as Lenmeldy, for the treatment of metachromatic leukodystrophy in pediatric patients. Lenmeldy is the first authorized gene therapy for children with pre-symptomatic late infantile or early juvenile metachromatic leukodystrophy (MLD), according to the FDA’s press release on the approval. Lenmeldy is also the first approved therapy for early juvenile or early symptomatic forms of the disease. Orchard CEO Bobby Gaspar in a statement said that Lenmeldy’s approval “opens up tremendous new possibilities” for children suffering from these early-onset forms of MLD, “who previously had no treatment options beyond supportive and end-of-life care.” The company on Monday did not provide specific launch details for Lenmeldy but promises to do so within the week. MLD is a rare and heritable metabolic disease that affects one in every 100,000 live births. It is characterized by a pathological build-up of sulfatides in the brain, liver, kidneys, spleen and other body parts, which over time can cause organ damage and neurological issues. Those with MLD often suffer from motor, behavioral and cognitive problems, as well as seizures. Patients also slowly lose the ability to move, talk, swallow and eat. Children with MLD often degenerate to a vegetative state and require round-the-clock care. Most patients die within five years of disease onset. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene, which under healthy circumstances produces an enzyme that breaks down sulfatides. Orchard’s Lenmeldy is an autologous gene therapy that uses a patient’s own stem cells to deliver a functioning copy of the ARSA gene. This mechanism of action won Lenmeldy the FDA’s Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations. Lenmeldy was also approved by the European Union in December 2020, where it is marketed as Libmeldy. The FDA’s approval of Lenmeldy is backed by data from two single-arm, open-label trials enrolling a total of 37 pediatric MLD patients. Over a median follow-up of 6.76 years, Lenmeldy led to significantly better overall survival compared with natural history data. The gene therapy also preserved motor and cognitive function in late-infantile MLD patients, whereas untreated counterparts had severe impairments. In terms of safety, Lenmeldy’s most common adverse events were febrile neutropenia, stomatitis, respiratory tract infections and rashes. These are reflected in the therapy’s label, which also bears warnings for thrombosis and thromboembolic events, encephalitis and the risk of hypersensitivity reactions. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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 .