Latest news with #CLN2
Yahoo
7 days ago
- General
- Yahoo
Swindon family of girl with rare disease 'horrified' at latest trial update
The Swindon family of a young girl with a rare brain disease welcomed the news that her trial of lifesaving treatment will continue indefinitely. Addy Clarke is just one of 100 children in the world to be diagnosed with Batten Disease (CLN2), which is gradually taking away her ability to talk, eat and walk independently, and see. Her parents Hayley and Dave have previously spoken to the Adver about their heartbreak at seeing their daughter fade before their eyes while she is supported by vital cerliponase alfa treatments. Earlier this month, they celebrated NICE and the NHS coming to a commercial agreement to continue that treatment for Addy and all other children with CLN2 after the trial ends in 2025, describing the news as 'amazing'. But they were saddened to learn that the same access to the treatment would not be given to any child diagnosed after January 1, 2026. Recommended reading Building 700 homes at old golf course 'best option' for the area Family pays tribute to cyclist killed in collision by Wiltshire driver Wedding venue can continue to host events after planning appeal NICE explained that although its committee took into account the condition's rarity, severity and the effect of the treatment on quality and length of life, the most likely cost-effective estimate based on the proposed price of the medicine is not within what it considers to be an acceptable use of NHS resources. In a statement on Addy's Batten Adventure Facebook page, the Clarke family said: 'Whilst we are happy for our own little Addy, we sadly know firsthand the devastation that this news can cause on a family - which would be made all the worse if you knew other children in the country were on the same treatment that your child couldn't access because of the timing they were diagnosed, and cost. 'For us, this treatment has never been simply just about our Addy, but all children diagnosed with this earth-shattering disease - therefore we will fight on. 'The decisions by NICE have been horrific and it feels like they are trying to manipulate the pharmaceutical companies by using families as pawns in game. 'Thank you for all your support you have shown us through the ongoing questions, the donations, prayers, meals and many other things over the past five years of this journey. 'We take a big deep breath, gather our thoughts and go again.' The family has met with South Swindon MP Heidi Alexander as they try to raise awareness of this disparity in the treatment of children with the rare disease. As for Addy herself, she recently celebrated her ninth birthday by having a party with her friends and classmates at Swindon's hydrotherapy pool.
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.
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
Fox Sports
06-05-2025
- Automotive
- Fox Sports
MSR Supporting Helen's Pink Sky Foundation
INDYCAR Meyer Shank Racing announced Tuesday its pledge to long-term support to Helen's Pink Sky Foundation, with its NTT INDYCAR SERIES cars driven by Felix Rosenqvist and Marcus Armstrong will carry the Foundation's decal in every race moving forward — and will do so until a cure is found for Batten disease. The pledge is aimed at driving awareness of CLN2 and Batten Disease which is a rare neurodegenerative disorder that affects just 2-4 of 100,000 children. This initiative holds a deeply personal significance for the MSR team and family. Three year old Helen Betty Born, the Foundation's namesake and relative of the Meyer family was born on May 31st, 2021 — the same day Helio Castroneves captured his historic fourth Indianapolis 500 victory with Meyer Shank Racing. Helen was diagnosed with Batten disease on March 10, 2025. Through devastation and heartache upon diagnosis, Helen pointed toward the pink sky which gave the Born family a sign of hope and a newfound purpose to find a cure for their little girl. "This one really hits close to home for us and we want to drive as much awareness as possible for this awful disease,' said Tim Meyer, MSR COO and Foundation Board Member. 'We're going to tell Helen's story everywhere we race and do our part to help this amazing little girl. Helen is so full of life and we just find her courage so inspiring. What she now faces is evil, it breaks my heart to see a child have to take on so much. We're going to fight like hell to help her and her family in anyway we can.' Together, MSR and the entire Born family will strive to create as much awareness as possible in hopes that a cure is found soon. "The response of the MSR team and their willingness to drive awareness for our little girl is so meaningful to me, our family and this foundation,' said Stephanie Born, Helen's Mom and Foundation Board Member. 'Our world changed dramatically not long ago and our journey ahead is a long and challenging one, but we won't lose hope, especially when it's all we have. Thank to Jim [Meyer], Mike [Shank], Tim [Meyer] and the entire team from the bottom of our heart, your support today and going forward will bring a smile to Helen's face.' Helen's Pink Sky Foundation will have its first activation May 30th-June 1st in honor of Helen's fourth birthday. The Foundation will host and encourage people around the country to host a pink lemonade stand with all proceeds going towards Batten disease research and finding a cure for Helen. For more information on how you can set up your own pink lemonade stand to honor Helen, visit recommended
