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Clinical setback in CNS rare disease
Ultragenyx’s Phase 3 program for Angelman syndrome delivered an outcome investors had been waiting for—and it wasn’t the one the company needed. The biotech said its antisense oligonucleotide...
Immunology: CAR-T safety spotlight
Novartis CAR-T trial disclosures have put fresh scrutiny on autoimmune risk after three deaths were reported in the company’s studies. The incidents are reigniting questions about what level of...
Oncology & molecular imaging premetastatic detection
A new review argues that tumor-derived extracellular vesicles (EVs) could be engineered into imaging tools to visualize the premetastatic niche before secondary tumors become detectable. The...
Immunotherapy deals: Roche Simcere trispecific licensing
Roche has struck an exclusive global licensing agreement with Simcere Zaiming for SIM-0660, a preclinical trispecific T-cell engager targeting CD79a/CD19/CD3. The deal includes a $75 million...
Manufacturing and regulatory science: EU animal-test replacement pilot
The EMA has launched a pilot program allowing companies to submit data generated by new approach methodologies (NAMs) for consideration outside the context of a formal marketing application. The...
Biopharma policy: FDA scrutiny of foreign data
Top FDA leaders are projecting enhanced scrutiny of clinical trial data generated outside the US, as lawmakers and industry attempt to respond to concerns about cross-border evidence use. The...
Diagnostics coverage expansion
Guardant Health said Carelon Healthcare Services will cover the Guardant Shield blood test for colorectal cancer screening starting Nov. 15. The policy update targets people at average risk above...
Regulatory action: Tavneos withdrawn by UK and EU
The MHRA is following the EMA by withdrawing Amgen’s Tavneos (avacopan) and banning new patient use as of Sept. 1. Supplies will remain available for about six months to help vasculitis patients...
ARPA-H funding: automated custom RNA medicines
ARPA-H awarded up to $125 million under its GIVE program to build a domestic, automated network for producing individualized, on-demand RNA-based genetic medicines with testing capacity near the...
Angelman syndrome clinical failure
Ultragenyx said its antisense oligonucleotide therapy GTX-102 for Angelman syndrome failed to show benefit in a Phase 3 trial, dealing a major blow to the company’s late-stage pipeline. The...
Huntington gene therapy seeks FDA priority review
UniQure moved a major regulatory step forward in Huntington’s disease by submitting applications to the FDA and the UK’s MHRA for AMT-130, a gene therapy the company says could become the first to...
Teva’s celiac disease gluten challenge results
Teva reported that its investigational antibody TEV’408 succeeded in a mid-stage celiac disease gluten challenge study by significantly reducing intestinal damage. In the trial, participants ate...
Roche expands B-cell franchise with Simcere trispecific TCE deal
Roche agreed to license Simcere Zaiming’s preclinical trispecific T-cell engager SIM-0660 in a deal worth up to $1.53 billion, with a $75 million upfront payment. The asset targets CD79a/CD19 on B...
Novartis pushes remibrutinib into MS Phase 3 wins
Novartis reported success for its BTK inhibitor remibrutinib (Rhapsido) in two large Phase 3 multiple sclerosis studies, describing superiority over Aubagio in reducing annualized relapse rates...
CAR-T safety sparks new debate after Novartis deaths
Novartis’ CAR-T trials have disclosed three deaths tied to autoimmune risk questions, Reuters-style prompting scrutiny of the therapy’s safety profile. The developments reportedly follow concerns...
ARPA-H funding for automated custom RNA manufacturing
ARPA-H awarded up to $125 million to fund the GIVE program, aiming to build a domestic, automated network for producing individualized, on-demand RNA-based genetic medicines. The effort will...
FDA and MHRA move against Amgen’s Tavneos
The UK’s MHRA joined the EMA in withdrawing Amgen’s Tavneos (avacopan) from the market and banning new patient use as of Sept. 1, with limited continued supply for existing patients. MHRA said...
Clinical trial operations: new benchmarking for trial sites
A new benchmarking framework aims to help clinical trial sites identify operational inefficiencies by standardizing evaluation across 36 performance benchmarks. The tool, developed from expert...
Planned recombinant manufacturing optimization for antibody cell lines
Sanofi researchers reported a vector engineering strategy designed to improve stable cell line production for multicistronic monoclonal antibodies using piggyBac transposon technology. The...
Clinical failure in rare disease
Ultragenyx disclosed that GTX-102 failed to demonstrate benefit versus sham in a large Phase 3 trial of Angelman syndrome, dealing a blow to a rare-disease program that had produced strong early...