Search Results
Jun 24, 2025, 09:25 ET Genprex Collaborators Present Positive Preclinical Research on Diabetes Gene Therapy at the 2025 American Diabetes Association 85th Scientific Sessions
(NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators presented positive preclinical research from studies of GPX-002, the Company's diabetes gene therapy drug candidate, at
More news about: Genprex, Inc.
Jun 24, 2025, 09:00 ET BioMarin Presents Five-Year Phase 3 Results Reinforcing Long-Term Efficacy and Safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2025 Congress
adoption of this gene therapy so they can experience the benefits that this treatment can offer." Below are key presentations for BioMarin and ROCTAVIAN at ISTH (all times Eastern Daylight Time): Efficacy, Safety and Quality of Life 5 Years After Valoctocogene Roxaparvovec Gene TransferPoster
More news about: BioMarin Pharmaceutical Inc.
Jun 24, 2025, 09:00 ET Supercede Therapeutics Provides Update on Novel Oral Treatment for Obesity
receptor type II (ACTRII) to treat obesity. ACTRII responds to ligands in the TGFb superfamily, including Activin, Myostatin, and Inhibin E, and induces gene expression changes in multiple tissues including adipose, liver, and muscle. Inhibition of the ACTRII signaling pathway has been shown in Phase II clinical
More news about: Supercede Therapeutics Inc.
Jun 24, 2025, 09:00 ET Nominations Open for 13th Annual International Harrington Prize for Innovation in Medicine
immunology.2018: Helen H. Hobbs, MD, UT Southwestern Medical Center, for the discovery of the link between a gene mutation (PCSK9) and lower levels of LDL, which has improved the treatment of high cholesterol.2017: Jointly awarded to Daniel
More news about: Harrington Discovery Institute
Jun 24, 2025, 08:15 ET Children's Hospital of Philadelphia and St. Jude Researchers Find Genetic Ancestry Influences How Gene Mutations Impact Cancer Prognosis in Patients With T-cell Acute Lymphoblastic Leukemia
patients have high risk or low risk disease. While previous studies found that genetic ancestry affects how certain gene changes appear in cancer, researchers can now show that these gene changes may also predict outcomes differently depending upon an individual's ancestry. "Our research demonstrates
More news about: Children's Hospital of Philadelphia
Jun 24, 2025, 07:00 ET Ascidian Therapeutics Appoints Dr. Murray A. Abramson as Chief Development Officer and Establishes Ophthalmology Clinical Advisory Board to Advance ACDN-01 for Treatment of Stargardt Disease
Stargardt disease – are examples of genetic disorders that cannot be addressed by standard gene replacement, given the large size of the gene, or by base editing, due to the high mutational variance of the affected gene. ACDN-01 is an in vivo RNA exon editor delivered by a single vector. It has demonstrated
More news about: Ascidian Therapeutics
Jun 24, 2025, 07:00 ET Bio-Techne and USP Announce Collaboration to Accelerate Monoclonal Antibody and Gene Therapy Product Development
throughout mAb development and manufacturing processes to demonstrate product safety and effectiveness. Separately, gene therapy, which relies on recombinant AAV to deliver gene edits into cells, is one of the fastest-growing sectors in the biopharmaceutical industry. The exceptional growth of these
More news about: Bio-Techne Corporation
Jun 24, 2025, 05:00 ET Klotho Neurosciences, Inc. and the Okinawa Research Center for Longevity Science, Leading Experts on the Okinawa "Blue Zone", Announce a Plan to Study Tissue Levels of the Human Klotho Gene and Protein in the World's Longest-Lived Population
KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and it's novel delivery systems to transform and improve the treatment of neurodegenerative and
More news about: Klotho Neurosciences, Inc.
Jun 24, 2025, 05:00 ET Klotho Neurosciences, Inc. and the Okinawa Research Center for Longevity Science, Leading Experts on the Okinawa "Blue Zone", Announce a Plan to Study Tissue Levels of the Human Klotho Gene and Protein in the World's Longest-Lived Population
KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and it's novel delivery systems to transform and improve the treatment of neurodegenerative and
More news about: Klotho Neurosciences, Inc.
Jun 24, 2025, 02:00 ET Nippon Shokubai Plans to Expand Its GMP-compliant Nucleic Acid Drug API Manufacturing Capacity Tenfold
manufacturing and quality control of pharmaceuticals (*2) Source: National Institute of Health Sciences. (n.d.). Division of Molecular Target and Gene Therapy Products, Section 2. Retrieved June 17, 2025, from
More news about: NIPPON SHOKUBAI CO., LTD.
Jun 23, 2025, 20:54 ET BrightGene Menyajikan Data Fase 2 yang Positif untuk Agonis Ganda GLP-1R/GIPR untuk Pengelolaan Berat Badan dan Diabetes Tipe 2 serta Data Praklinis untuk Analog Amylin Baru di Sesi Ilmiah ke-85 Asosiasi Diabetes Amerika
abetes-tipe-2-serta-data-praklinis-untuk-analog-amylin-baru-di-sesi-ilmiah-ke-85-asosiasi-diabetes-amerika-302488867.html SOURCE Bright Gene
More news about: Bright Gene
Jun 23, 2025, 20:54 ET BrightGene Presents Positive Phase 2 Data for Dual GLP-1R/GIPR Agonist for Weight Management and Type 2 Diabetes and Preclinical Data for Novel Amylin Analog at American Diabetes Association's 85th Scientific Sessions
es-and-preclinical-data-for-novel-amylin-analog-at-american-diabetes-associations-85th-scientific-sessions-302488865.html SOURCE Bright Gene
More news about: Bright Gene
Jun 23, 2025, 11:30 ET Actinium Highlights Expanded Data Set for ATNM-400 in Prostate Cancer Demonstrating Superior Efficacy to Enzalutamide and Ability to Overcome Resistance to ARPI Therapy and PSMA-Ac-225/Lu-177 Labelled Radiotherapy at the Society of Nuclear Medicine & Molecular Imaging Annual Meeting
next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with r/r AML, which Actinium is seeking a potential
More news about: Actinium Pharmaceuticals, Inc.
Jun 23, 2025, 09:05 ET Cumberland Pharmaceuticals Shares Phase 2 FIGHT DMD Trial Results at the Parent Project Muscular Dystrophy Annual Conference
www.fightdmdtrial.com About Duchenne Muscular Dystrophy (DMD) DMD is a rare and incurable pediatric disease caused by mutations in the gene encoding dystrophin, a protein critical for muscle function, including the heart. Patients with DMD slowly lose muscle function, resulting in the inability
More news about: Cumberland Pharmaceuticals Inc.
Jun 23, 2025, 09:00 ET Genprex Collaborators Present Research on Non-Viral Approach to Diabetes Gene Therapy Using Lipid Nanoparticle Delivery System at the 2025 American Diabetes Association 85th Scientific Sessions
GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company's diabetes gene therapy drug candidate,
More news about: Genprex, Inc.
Jun 23, 2025, 07:00 ET Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
About PhenylketonuriaPhenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe — an essential amino acid found in all
More news about: PTC Therapeutics, Inc.
Jun 22, 2025, 22:01 ET IASO Bio presents promising efficacy of Equecabtagene Autoleucel in patients with Multiple Sclerosis in IIT study at the 11th Congress of the European Academy of Neurology (EAN)
Autoleucel (Eque-cel) Equecabtagene Autoleucel (Eque-cel) is an innovative fully human anti-BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane, and 4-1BB co-stimulatory and CD3ζactivation
More news about: IASO Bio
Jun 22, 2025, 15:35 ET Mim8 prophylaxis treatment shown to be well-tolerated when switching from emicizumab in people with hemophilia A in new phase 3 data presented at the ISTH 2025 Congress
https://clinicaltrials.gov/study/NCT05685238.U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed May 2025. Available at
More news about: Novo Nordisk
Jun 22, 2025, 12:50 ET RCKT INVESTOR DEADLINE: Robbins Geller Rudman & Dowd LLP Announces that Rocket Pharmaceuticals, Inc. Investors with Substantial Losses Have Opportunity to Lead Class Action Lawsuit
[email protected]. CASE ALLEGATIONS: Rocket Pharmaceuticals operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. The Rocket Pharmaceuticals class action lawsuit alleges that defendants provided
More news about: Robbins Geller Rudman & Dowd LLP
Jun 22, 2025, 10:00 ET INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Rocket Pharmaceuticals, Inc. - RCKT
action] On May 27, 2025, Rocket issued a press release "announc[ing] an update related to RP-A501, its investigational gene therapy for Danon disease." The press release disclosed that "[a] patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected
More news about: Pomerantz LLP
Jun 22, 2025, 10:00 ET Pomerantz Law Firm Announces the Filing of a Class Action Against PepGen Inc. and Certain Officers - PEPG
dystrophy ("DMD"), a genetic disorder characterized by progressive muscle degeneration and weakness. DMD is caused by the mutation of the dystrophin gene, resulting in, inter alia, a limited production of the dystrophin protein, which in turn leads to DMD's clinical features. According to PepGen,
More news about: Pomerantz LLP
Jun 21, 2025, 09:00 ET MetaVia Presents Pre-Clinical Data on DA-1241 Demonstrating Additive Hepatoprotective Effects in Combination With Efruxifermin at the ADA's 85th Scientific Session
marked decreases in inflammatory (TNFα -58%, CXCL10 -56%, CCL2 -77%, galectin-3 -87%) and fibrotic (type 1 collagen α1 -72%, α-SMA -59%, TIMP1 -88%) gene expression in the combination. Notably, hedgehog-interacting protein, a suppressor of hepatic stellate cell activation, was upregulated more (+321%)
More news about: MetaVia Inc.
Jun 21, 2025, 08:56 ET New data demonstrates robust efficacy of Vyepti® (eptinezumab) in otherwise difficult-to-treat patients with severe migraine
mainly from European countries. About Vyepti® (eptinezumab)Eptinezumab is a humanized monoclonal antibody that binds to calcitonin gene-related peptide (CGRP) which was intentionally designed for IV administration. The efficacy and safety of eptinezumab was evaluated in two phase III
More news about: H. Lundbeck A/S
Jun 21, 2025, 08:54 ET New data demonstrates robust efficacy of Vyepti® (eptinezumab) in otherwise difficult-to-treat patients with severe migraine
mainly from European countries. About Vyepti® (eptinezumab)Eptinezumab is a humanized monoclonal antibody that binds to calcitonin gene-related peptide (CGRP) which was intentionally designed for IV administration. The efficacy and safety of eptinezumab was evaluated in two phase III
More news about: H. Lundbeck A/S
Jun 21, 2025, 08:40 ET New data from phase III trial confirms efficacy of Vyepti® (eptinezumab) in Asian population with chronic migraine
(IV) infusion.2 About Vyepti® (eptinezumab) Eptinezumab is a humanized monoclonal antibody that binds to calcitonin gene-related peptide (CGRP) which was intentionally designed for IV administration. The efficacy and safety of eptinezumab was evaluated in two phase III
More news about: H. Lundbeck A/S