LONDON, UK — Spur Therapeutics today announced updated clinical data for avigbagene parvec (FLT201), its Gaucher disease gene therapy candidate, demonstrating clinical benefit and favorable safety maintained for more than two years to date following administration of a single low dose. These data are being presented at the European Society of...
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STEVENAGE, UK – FLT201, an experimental gene therapy developed by Spur Therapeutics, has shown sustained clinical benefits for up to 21 months in people with Gaucher disease type 1, according to new data from a clinical trial. The findings, presented at the 28th Annual Meeting of the American Society of...
ALISO VIEJO, Calif. — SpyGlass Pharma™, a privately-held ophthalmic biotechnology company, announced today 1-year follow up data from a first-in-human study of 23 patients with glaucoma or ocular hypertension implanted with SpyGlass’ Intraocular Lens (IOL) based Drug Delivery Platform with bimatoprost at the time of cataract surgery. These data will...
Mill Valley, CA – SRF has established a dedicated account to support research regarding SYNGAP1 missense variants. This account is being launched with a $10,000 donation from Dennis and Janet Nordmoe. This is a cause close to their hearts, as their granddaughter Olivia was diagnosed with SYNGAP1-Related Disorder (SRD) caused...
Barcelona, Spain – New research being presented at this year’s ESCMID Global Congress (formerly ECCMID) in Barcelona, Spain (27-30 April) has found compelling evidence that tuberculosis (TB) can have a lasting impact on the lungs of individuals who have been successfully treated for the disease. TB survivors have smaller lungs...
MEMPHIS, Tenn. – Scientists at St. Jude Children’s Research Hospital comprehensively identified genes directly regulated by a protein associated with high-risk pediatric leukemias. High-risk leukemias, particularly MLL-rearranged (MLL-r) leukemia, often overexpress the homeodomain transcription factor HOXA9 protein, which cannot currently be targeted with drugs. This study provides a foundation for...
FLORISSANT, Mo. – After a viral video on social media, one St. Louis girl got to live her dream of owning a bakery for the day. On top of that, customers raised enough money for her to have a once in a lifetime treatment, that increases her ability to live....
Guangzhou, China – Researchers from the South China University of Technology identified a novel approach to effectively silence the expression of transthyretin (TTR), with potential implications for innovative therapeutic approaches in TTR amyloidosis, including transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The proposed strategy involves stabilizing the G-quadruplex structure—a fundamental genomic structural feature—and...
The burden from activating KIT proto-oncogene (KIT) mutations in patients with advanced systemic mastocytosis (advSM) can be relieved with KIT inhibitors, improving the clinical trajectory of the disease. The protocols for determining staging of SM require updating since these drugs have become available, according to a review published in the International...
Stanford, Calif. – An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a phase 1 clinical trial has shown. While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem cell transplant...