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Glioblastoma Exploits Gasdermin E for Pyroptosis Resistance
Contradicting conventional understanding, Gasdermin E (GSDME), a known pyroptosis mediator, paradoxically enables glioblastoma cells to resist pyroptotic death while promoting tumor progression....
Vertex Stem Cell Therapy Enables Insulin Independence in Diabetes
Vertex Pharmaceuticals’ investigational allogeneic stem cell therapy for type 1 diabetes has demonstrated promising clinical results, enabling 10 out of 12 participants to discontinue insulin...
Novo Nordisk’s Amylin Drug Shows Potent Weight Loss but Dosing Questions Remain
Novo Nordisk's phase 1/2 study of amycretin, a next-generation obesity amylin analogue, demonstrated substantial weight loss exceeding 24% at the highest dose after 36 weeks. However, weight...
FDA Pilots Priority Review to Accelerate Biotech Approvals
The FDA, under Commissioner Martin Makary, has launched a pilot commissioner’s national priority voucher program designed to shorten review times for biotech drugs by one to two months. The...
Capricor’s Deramiocel Shows Four-Year Efficacy in Duchenne Muscular Dystrophy
Capricor Therapeutics announced positive four-year data from its HOPE-2 open-label extension trial of Deramiocel, a cell therapy for Duchenne Muscular Dystrophy. Presented at the PPMD 2025 Annual...
CHMP Recommends Five New Drugs Including Madrigal’s NASH Treatment
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of five new medicines, including Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) for...
Nrf2-HMOX1 Axis Unlocks Lung Cancer Chemotherapy Resistance
Researchers have identified the Nrf2-HMOX1 pathway as a key mediator of cisplatin resistance in non-small cell lung cancer (NSCLC), unveiling a mechanism that cancer cells use to evade...
Fanconi Anemia Treated with Translational Read-Through Drugs
A novel class of translational read-through-inducing drugs (TRIDs) has been developed to address nonsense mutations in Fanconi anemia, a genetic disorder causing bone marrow failure and cancer...
Vertex’s Type 1 Diabetes Cell Therapy Shows Sustained Insulin Independence
Vertex Pharmaceuticals announced promising results from its stem cell-derived therapy for type 1 diabetes, with 10 of 12 patients able to discontinue insulin injections a year post-infusion. The...
Novo Nordisk’s Amycretin Obesity Drug Data Raises Dosing Questions
Novo Nordisk’s Phase 1/2 trial of amycretin, a next-generation amylin-targeting obesity injection, demonstrated substantial weight loss across multiple dose levels. However, overlapping efficacy...
Artificial Intelligence Diagnoses Vocal Cord Paralysis Severity
Researchers have developed a novel AI platform employing convolutional neural networks and Mel-spectrogram analysis to objectively stage unilateral vocal cord paralysis (UVCP). The model...
Prothena Cuts 63% Workforce Amid Amyloidosis Drug Failure
Biotech firm Prothena announced a workforce reduction affecting 63% of employees following the failure of its amyloidosis drug birtamimab in a Phase 3 trial for Mayo stage IV AL amyloidosis....
FDA Unveils Pilot Program to Expedite Drug Reviews
On the opening day of the 2025 Biotechnology Innovation Organization convention, FDA Commissioner Martin Makary announced a new pilot initiative granting selected companies access to a...
Syncona Realigns Fund Amid Biotech Market Downturn
The UK-based life sciences investment firm Syncona announced a strategic restructuring and a shift toward establishing a new evergreen fund vehicle, aiming to navigate the ongoing bleak biotech...
Michael J. Fox Foundation Launches Parkinson’s Therapeutic Target Program
The Michael J. Fox Foundation initiated the Targets to Therapies (T2T) program, a collaborative, community-driven effort to accelerate validation of 21 promising biological targets for Parkinson’s...
CHMP Recommends Approval of Multiple New Drugs in Europe
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended authorization for five new drugs, including conditional approval of Madrigal Pharmaceuticals’...
Nrf2-HMOX1 Axis Controls Cisplatin Resistance in Lung Cancer
Researchers have identified the Nrf2-HMOX1 pathway as a key mediator of cisplatin resistance in non-small cell lung cancer (NSCLC). This axis suppresses ferroptosis, an iron-dependent form of cell...
Novo Nordisk's Amycretin Obesity Drug Raises Dosing Questions
Novo Nordisk's experimental obesity drug amycretin, targeting the amylin hormone, demonstrated significant weight loss in early clinical trials but showed similar efficacy across different dosing...
Vertex’s Stem Cell Therapy Enables Insulin Independence in Type 1 Diabetes
Vertex Pharmaceuticals presented promising data indicating that their off-the-shelf stem cell therapy allowed 10 of 12 type 1 diabetes patients to discontinue insulin use after one year. The...
CHMP Recommends New Drugs and Reviews Alzheimer's Therapy
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended five new medicines this week, including conditional approval of Madrigal Pharmaceuticals’...