Lexeo Therapeutics’ Post

AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery!!! This could be an important step in the next phase of CNS therapeutic development. https://lnkd.in/eS7JeTSD

Ophthopedia Update:Positive early safety, efficacy data reported for Leber congenital amaurosis gene therapy: Atsena Therapeutics reported positive 12-month safety and efficacy data from a phase 1/2 trial of ATSN-101 gene therapy in GUCY2D-associated Leber congenital amaurosis. Fifteen patients received unilateral subretinal injections of ATSN-101. The trial included three adult cohorts (three patients each) that received ascending doses. One adult cohort and one pediatric cohort (three patients each) subsequently received a high dose of the therapy, with nine patients in total receiving the high dose. There were no serious treatment-emergent adverse events at 12 months, according to a press release,

#Fast Track Designation for #ATA-100, an AAV-based gene therapy for the treatment of LGMD-R9 LGMD-R9 in #Phase 1/2 clinical trials in the US and EU. The first low-dose cohort of the study in EU shows promising initial functional results and two patients have already been treated in the high-dose cohort. Initial results show promise, with no unexpected safety issues. Updated results of the clinical trials are expected in Oct 2024.

Lysosomal storage diseases (LSDs) are progressive disorders that can affect tissues and organs throughout the body, the central nervous system (CNS), and body systems like the skeletal system. With enzyme replacement therapy, providers can address somatic symptoms but cannot treat CNS symptoms because the blood-brain barrier (BBB) prevents it. Hiroyuki Sonoda, Head of Research at JCR Pharmaceuticals, recently presented promising data that showed the potential for gene therapy, in combination with JCR’s J-Brain Cargo® technology, to address CNS symptoms. At the 7th International Lysosomal Disease Forum Executive Committee, Sonoda illustrated how the J-Brain Cargo® technology can be applied to gene therapy to cross the BBB and effectively deliver biologics into the brain. J-Brain Cargo® is already leveraged in an approved treatment for MPS II in Japan, but this new application presents potential for a gene therapy approach to treat CNS diseases that are inaccessible by existing approaches. For more about the potential of J-Brain Cargo®, click here: https://bit.ly/3SALcsV. #RareDisease #BloodBrainBarrier #LysosomalStorageDisorders #JCRPharma

Gene therapy has undergone technological enhancements over the last 20 years that have transformed this field invigorating the development of many novel and potentially life-changing therapeutics. #Pain #PainManagement #BetterSolutions #PainSolution #PainRelief #GeneTherapy

U.K. has approved CASGEVY, a pioneering CRISPR-based gene therapy by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating sickle cell disease and beta-thalassemia. This marks a significant milestone, as it offers a one-time treatment potentially curing these genetic conditions, which previously required ongoing blood transfusions or bone marrow transplants with a risk of rejection. CASGEVY works by enhancing fetal hemoglobin production, compensating for the defective adult hemoglobin in patients. This approval not only represents hope for those affected but also opens avenues for the growth of other Life Science companies as it builds investors eyes back. What else have you seen in Life Science that motivates you for 2024?

Excellent news and good to see the IMF finally being used as was originally intended. Perhaps this breakthrough deal with CSL Behring's Hemgenix (haemophilia B) will now give industry more confidence and an 'art-of-the-possible' mindset in order to create more managed access for promising new non-cancer medicines. #access #health and access to medicines # pricing # value # IMF # NICE # NHS commercial framework for new medicines

Exciting developments are happening in the world of Cell and Gene Therapy (CGT)! 🔬 The number of life-changing CGT launches in the UK are projected to rise significantly, over the next year and beyond! 🚀 However, complex care pathways, in addition to limited knowledge and skills around this therapy-area, threaten to diminish the impact of CGT launches. 🛑 Discover how Patient Support Programmes (PSPs) can effectively address these issues and contribute to the success of CGT therapies, here 👉 https://lnkd.in/ebCJNEgK #Healthcare #Pharmaceuticals #GeneTherapy #NHS #PatientCare #PatientSafety #PatientExperience #PatientCentricity

There are now two gene therapies for the treatment of Hemophilia B. They are designed to reduce both bleeding episodes and the need for prophylactic therapy – at a cost of $3.5 million for a one-time dose. Read our Pharmacy Focus article to learn more about Hemophilia B and its treatment: https://lnkd.in/e2vcW9Ua

FDA Approves CRISPR-Based Gene Therapy Casgevy To Treat Beta Thalassemia CNN (1/16, Goodman) reports the FDA “has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.” Now, “the groundbreaking treatment can...also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder.” The therapy’s price is set at “$2.2 million for both sickle cell disease and beta thalassemia.” https://buff.ly/48BO3I0