IBEC researchers led by Javier Ramón and Juan M. Fernández develop the first three-dimensional model for myotonic dystrophy, a rare disease that currently has no cure. The model combines patient cells and bioengineering techniques and represents a major advance over the use of animals and cell cultures. This new model...
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SAN DIEGO, Calif. — Tr1X, a clinical-stage biopharmaceutical company developing first-in-class allogeneic engineered type 1 regulatory (Tr1 Treg) and CAR-Tr1 Treg cell therapies with the potential to cure autoimmune and inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND)...
Over 100,000 people suffer from multiple sclerosis in Germany alone. Despite intensive research, the factors that trigger the disease and influence its progress remain unclear. Scientists from the Max Planck Institute of Neurobiology in Martinsried and an international research team have succeeded in attaining three important new insights into the...
BOSTON — Tract Bio, a biotechnology company discovering and developing novel therapies for cancer and inflammatory disease, today announced the publication of a research article in the American Journal of Respiratory and Critical Care Medicine (AJRCCM) highlighting the stem cell variants driving chronic lung inflammation in modulator-treated cystic fibrosis patients...
COLUMBIA, Missouri – As one of the most insidious diseases in the world, cancer has few treatments that work to eradicate it completely. Now, a new ground-breaking approach pioneered by two researchers working at the University of Missouri’s Roy Blunt NextGen Precision Health building shows promising results in preventing lung...
Bottom Line: The MEK inhibitor trametinib (Mekinist) was an effective treatment for pediatric patients with relapsed or refractory juvenile myelomonocytic leukemia (JMML) enrolled in a phase II clinical trial, with seven of 10 patients alive after a median of two years. Journal in Which the Study was Published:...
SAN DIEGO, Calif. — Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to TPN-101 for progressive supranuclear palsy (PSP). “Fast...
NANJING, China and GAITHERSBURG, Md. — TransThera Sciences Inc. (“TransThera”) announced the publication of clinical results from a US-based Phase 2 trial evaluating tinengotinib in patients with Cholangiocarcinoma (CCA) on The Lancet Gastroenterology and Hepatology (Impact Factor: 38.6). Cholangiocarcinoma (CCA) is an aggressive malignancy of the bile ducts, frequently driven by FGFR2 fusions-genomic...
SAN DIEGO, CA — Travere Therapeutics Inc. (Nasdaq: TVTX) today announced the completion of a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) in IgA nephropathy (IgAN). The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for...
SAN DIEGO — Travere Therapeutics, Inc. (NASDAQ: TVTX) announced that the interim analysis of efficacy and safety data from the ongoing Phase 3 PROTECT Study evaluating FILSPARI™ (sparsentan) in IgA nephropathy (IgAN) will be presented in late-breaking trial sessions at both the ISN World Congress of Nephrology in Bangkok, Thailand,...