Latest news with #LSDs
Yahoo
06-06-2025
- Business
- Yahoo
JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website
- A refreshed global brand signals JCR's expanded international presence and unwavering focus on rare and genetic diseases worldwide - HYOGO, Japan, June 06, 2025--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the launch of its new global website. As the company turns 50, it is marking its growing footprint in Japan, the U.S., Europe, and Latin America with a unique website tailored for global audiences that reflects its passion for both science and humanity and brings the JCR brand to life for patients, physicians, and partners worldwide. "At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world," said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. "In September, we celebrate the 50th anniversary of the company I founded in 1975. The launch of our new global website reflects our evolution from an innovative startup in Japan to a global biopharmaceutical company. We remain dedicated to developing therapies for people with rare and genetic diseases and partnering with those who share our vision of transforming care for patients and families living with once untreatable diseases." The site features streamlined user interface and curated content for patients, physicians, partners, and investors – reinforcing JCR's commitment to bridge the gap between complex science and unmet needs of patients and families. For the past 50 years, JCR has built a reputation for the development of innovative new therapeutic approaches, including its more recent J-Brain Cargo® technology, a proprietary blood-brain barrier-penetrating technology that can deliver biotherapeutics into the central nervous system (CNS). This platform underpins several investigational pre-clinical and clinical staged therapies in JCR's pipeline, beginning with lysosomal storage disorders (LSDs), and it reflects the company's ongoing commitment to advancing treatments for CNS-related diseases across neurodegeneration, neuro-inflammation, and neuro-oncology conditions. JCR continues to strengthen its global research, development, and manufacturing capabilities, with a focus on addressing unmet medical needs and expanding therapeutic possibilities. Learn more about JCR, its technologies, and clinical development programs on its global website: About the J-Brain Cargo® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO® (INN: pabinafusp alfa). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website: View source version on Contacts Investors & Media:JCR Pharmaceuticals Co., Communicationsir-info@ Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Business Wire
06-06-2025
- Business
- Business Wire
JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website
HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; 'JCR'), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the launch of its new global website. As the company turns 50, it is marking its growing footprint in Japan, the U.S., Europe, and Latin America with a unique website tailored for global audiences that reflects its passion for both science and humanity and brings the JCR brand to life for patients, physicians, and partners worldwide. 'At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'In September, we celebrate the 50 th anniversary of the company I founded in 1975. The launch of our new global website reflects our evolution from an innovative startup in Japan to a global biopharmaceutical company. We remain dedicated to developing therapies for people with rare and genetic diseases and partnering with those who share our vision of transforming care for patients and families living with once untreatable diseases.' The site features streamlined user interface and curated content for patients, physicians, partners, and investors – reinforcing JCR's commitment to bridge the gap between complex science and unmet needs of patients and families. For the past 50 years, JCR has built a reputation for the development of innovative new therapeutic approaches, including its more recent J-Brain Cargo ® technology, a proprietary blood-brain barrier-penetrating technology that can deliver biotherapeutics into the central nervous system (CNS). This platform underpins several investigational pre-clinical and clinical staged therapies in JCR's pipeline, beginning with lysosomal storage disorders (LSDs), and it reflects the company's ongoing commitment to advancing treatments for CNS-related diseases across neurodegeneration, neuro-inflammation, and neuro-oncology conditions. JCR continues to strengthen its global research, development, and manufacturing capabilities, with a focus on addressing unmet medical needs and expanding therapeutic possibilities. Learn more about JCR, its technologies, and clinical development programs on its global website: About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO ® (INN: pabinafusp alfa). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website:
Time of India
01-06-2025
- Health
- Time of India
Rare disease patients continue to struggle despite govt policies
New Delhi: Despite the establishment of the National Policy for Rare Diseases (NPRD) in 2021 and a nationwide financial assistance programme, over 300 patients, predominantly children diagnosed with Lysosomal Storage Disorders (LSDs) such as Gaucher, Pompe, Fabry, MPS I, and II, are left without essential medical care. Tired of too many ads? go ad free now Among them, 70 are from Delhi. Since 2022, nearly 50 patients—including six from Delhi—have succumbed to these diseases. The Lysosomal Storage Disorders Support Society (LSDSS) reports that 50 patients across India exhausted their one-time funding under the policy. In Delhi, 7 patients are in critical condition due to financial constraints. Alishba Khan, a young girl battling Gaucher disease, illustrates the dire consequences of limited funding. Her father, Maqsood Alam, recounts the devastating decline in her health following the discontinuation of her medication in Aug 2024. "She suffers from severe abdominal pain, an enlarged spleen, bone pain, weakness, and decreased haemoglobin level. She is unable to attend school and requires constant supervision," Alam shared, his voice filled with anguish. Having already lost four children, he continually appeals to the High Court for continued treatment, fearing for Alishba's survival. While doctors at AIIMS offer significant support, they are constrained by limited funding, he said. Recognising the critical need, the Delhi High Court on Oct 4, 2024, directed the Ministry of Health and Family Welfare to extend funding beyond the Rs 50 lakh cap for patients with rare diseases. The court also mandated the establishment of a Rs 974 crore national fund for rare diseases for the fiscal years 2024-25 and 2025-26. Tired of too many ads? go ad free now However, the Ministry challenged this directive in the Supreme Court, where the matter remains pending. The Lysosomal Storage Disorders Support Society (LSDSS) has been vigorously campaigning for urgent action. In a recent appeal to Union Health Minister JP Nadda, the LSDSS highlighted that despite allocating over Rs 205 crore to 12 Centres of Excellence (CoEs)—including AIIMS Delhi and MAMC—more than 300 eligible patients remain untreated, and at least 50 patients have died while awaiting treatment. LSDSS National President Manjit Singh emphasised that many of these patients are children with conditions for which DCGI-approved therapies exist. " In the Budget Session of Parliament, the Ministry reaffirmed that no eligible patient should be denied life-saving therapy. However, the disconnect between funding and actual treatment on the ground continues to undermine this commitment," the letter stated. The society requested the ministry to direct CoEs to immediately accept eligible patients, ensure compliance with NPRD 2021 guidelines, prioritise patients with approved therapies, establish a rapid response task force for timely treatment initiation, and authorise continued treatment support beyond the Rs 50 lakh cap based on clinical merit. Singh acknowledged the govt's historic support for rare disease patients, requesting leadership to ensure this support translates into timely, life-saving access. Officials at MAMC explained that the crowdfunding portal has technical constraints preventing status updates from 'waiting for treatment' to 'under treatment' after therapy begins. Although the portal displays zero patients receiving treatment, in reality, 10 individuals out of 28 registered cases of LSDs are currently undergoing enzyme replacement therapy (ERT). The administration confirmed that treatment allocation strictly follows approved eligibility standards, considering ERT's expected clinical effectiveness and requires approval from the Centre of Excellence (CoE) committee, comprising various medical specialists. They noted that patients with significant neurological symptoms receive lower priority, as current evidence suggests ERT offers minimal benefits in such instances. The focus remains on treating patients with a higher likelihood of substantial clinical benefits. Regarding the national policy's one-time financial support limit of Rs 50 lakh per patient, the MAMC officials indicated their inability to provide treatment beyond this amount without specific instructions from senior authorities. When contacted, Dr Neerja Gupta from the genetic unit of paediatrics at AIIMS said she doesn't have the details on this matter offhand.
Yahoo
14-05-2025
- Health
- Yahoo
Latus Bio Unveils AAV-Ep+ Capsid Variant Capable of Unprecedented Protein Production in the Brain
PHILADELPHIA, May 14, 2025--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, "AAVs engineered for robust brain transduction drive therapeutically relevant expression of secreted recombinant protein in NHPs and a mouse model of lysosomal storage disease." The study, led by Latus founder Beverly Davidson, PhD details the development of a novel adeno-associated virus (AAV) capsid variant - AAV-Ep+ - that demonstrates unprecedented potency in transducing cells that line the ventricles, known as ependymal cells, and cerebral neurons in mice and in non-human primates (NHPs). This advancement is potentially a significant leap forward for therapeutic gene delivery, wherein the study authors demonstrate that cells transduced with AAV-Ep+ can effectively serve as protein production depots, secreting large amounts of soluble proteins into the cerebral spinal fluid (CSF) for uptake throughout the central nervous system (CNS). This potency and distribution profile could potentially result in one-time delivery of gene therapies that encode protein treatments for lysosomal storage disorders (LSDs) as well as for other neurodevelopmental and neurodegenerative diseases that result in long term benefits for patients. The AAV-Ep+ capsid variant was identified through a massively parallelled and unbiased screen of a large-diversity AAV variant library administered to NHPs. The nominated capsid, which was isolated from tens of millions of potential candidates, displays: Remarkable tropism for cells that line the ventricular system of the brain and spinal cord of adult NHPs and mice. It also efficiently transduces neurons in cortical regions of the brain that are implicated in many diseases. Robust transduction of induced pluripotent stem cell (iPSC)-derived human neurons and mice when compared to naturally occurring AAV serotypes. This cross-species activity highlights the potential for AAV-Ep+ to deliver sustained and therapeutic expression of encoded proteins in human brain cells that could result in prolonged therapeutic benefit for patients. Low dose administration of AAV-Ep+ constructs designed to express human tripeptidyl peptidase (hTPP1) to mice deficient in TPP1 (a model of human CLN2 disease - a type of LSD) as well as to NHPs result in CSF and parenchymal tissue levels that exceeded those obtained with natural serotype capsids, reaching levels that are potentially multi-fold above therapeutic values required for CLN2 patients. "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. "AAV-Ep+ offers a highly efficient, low-dose solution for brain-wide protein delivery, opening new possibilities for treating neurodevelopmental diseases like CLN2 disease and beyond." The study showcases Latus' unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in other regions of the central nervous system (e.g., cortex, cerebellum, spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs. About Latus Bio (Latus) Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit and follow on LinkedIn. View source version on Contacts info@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
14-05-2025
- Health
- Business Wire
Latus Bio Unveils AAV-Ep+ Capsid Variant Capable of Unprecedented Protein Production in the Brain
PHILADELPHIA--(BUSINESS WIRE)-- Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, 'AAVs engineered for robust brain transduction drive therapeutically relevant expression of secreted recombinant protein in NHPs and a mouse model of lysosomal storage disease.' "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. The study, led by Latus founder Beverly Davidson, PhD details the development of a novel adeno-associated virus (AAV) capsid variant - AAV-Ep+ - that demonstrates unprecedented potency in transducing cells that line the ventricles, known as ependymal cells, and cerebral neurons in mice and in non-human primates (NHPs). This advancement is potentially a significant leap forward for therapeutic gene delivery, wherein the study authors demonstrate that cells transduced with AAV-Ep+ can effectively serve as protein production depots, secreting large amounts of soluble proteins into the cerebral spinal fluid (CSF) for uptake throughout the central nervous system (CNS). This potency and distribution profile could potentially result in one-time delivery of gene therapies that encode protein treatments for lysosomal storage disorders (LSDs) as well as for other neurodevelopmental and neurodegenerative diseases that result in long term benefits for patients. The AAV-Ep+ capsid variant was identified through a massively parallelled and unbiased screen of a large-diversity AAV variant library administered to NHPs. The nominated capsid, which was isolated from tens of millions of potential candidates, displays: Remarkable tropism for cells that line the ventricular system of the brain and spinal cord of adult NHPs and mice. It also efficiently transduces neurons in cortical regions of the brain that are implicated in many diseases. Robust transduction of induced pluripotent stem cell (iPSC)-derived human neurons and mice when compared to naturally occurring AAV serotypes. This cross-species activity highlights the potential for AAV-Ep+ to deliver sustained and therapeutic expression of encoded proteins in human brain cells that could result in prolonged therapeutic benefit for patients. Low dose administration of AAV-Ep+ constructs designed to express human tripeptidyl peptidase (hTPP1) to mice deficient in TPP1 (a model of human CLN2 disease - a type of LSD) as well as to NHPs result in CSF and parenchymal tissue levels that exceeded those obtained with natural serotype capsids, reaching levels that are potentially multi-fold above therapeutic values required for CLN2 patients. "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. "AAV-Ep+ offers a highly efficient, low-dose solution for brain-wide protein delivery, opening new possibilities for treating neurodevelopmental diseases like CLN2 disease and beyond." The study showcases Latus' unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in other regions of the central nervous system (e.g., cortex, cerebellum, spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs. About Latus Bio (Latus) Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit and follow on LinkedIn.
