Targeted Oncology18h
FDA Grants Orphan Drug Designation to MB-105 for R/R T-Cell Lymphoma
MB-105, a first-in-class CD5-targeted CAR T-cell therapy, received FDA orphan drug designation for relapsed/refractory T-cell ...
March Biosciences Receives FDA Orphan Drug Designation for MB-105, a First-in-Class CD5 CAR-T Cell Therapy, for T-Cell Lymphoma
Company prepares to advance to Phase 2 clinical trial in early 2025HOUSTON, Jan. 28, 2025 (GLOBE NEWSWIRE) -- March Biosciences (March Bio), an ...
SEED receives FDA Rare Pediatric Disease, Orphan Drug designations for ST-01156
SEED Therapeutics announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease and Orphan Drug designations to ...
TMCnet2d
US Patent Office Granted Can-Fite Namodenoson Patent for Use as anti-Obesity Drug
The patent application No. 17/309,952 entitled “An A3 adenosine receptor ligand for use for achieving a fat loss effect”, has been accepted by the US Patent Office, will be issued on February 2 nd and ...
Cure Today5d
FDA Approves GRAFAPEX/Fludarabine Before alloHSCT in AML and MDS
The FDA has approved GRAFAPEX™ and fludarabine as a preparative regimen in acute myeloid leukemia or myelodysplastic syndrome ...
Contract Pharma6d
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
ophthalmologytimes6d
FDA grants fast track designation to N-acetylcysteine amide tablets from Nacuity Pharmaceuticals
The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with ...
News Medical6d
What is an Orphan Drug? A Guide to Rare Disease Therapies
Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...
BioWorld7d
Tikun programs receive orphan, rare pediatric disease designations
Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the ...
Opinion
Column: Lawmakers should reject drug affordability board legislation
Virginia’s proposed prescription drug affordability board would positioning the government as an arbiter of medical value and access, the Rare Access Action Project’s Michael Eging ...
Nuvalent: A First Look
Nuvalent, Inc. shares have fallen approximately 30% after promising updates on its two lead NSCLC candidates rallied the ...
Hosted on MSN7d
Soligenix (NASDAQ: $SNGX) Unveils Two-Pronged Approach To Tackle Rare Diseases & Unmet Medical Needs
Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases and areas of unmet medical need, has a portfolio of products aimed at ...