Advanced Therapies
China's Belief BioMed presented promising new Phase III results at ASH for its AAV gene therapy for hemophilia B, although the wider China CGT space continues to be dominated by oncology.
UniQure said discussions with the FDA resulted in a path forward for seeking accelerated approval of its gene therapy AMT-130 in Huntington’s disease.
BMS presented Phase I data for arlocabtagene autoleucel, showing a strong median PFS and lower incidence and severity of trouble swallowing, a common side effect of anti-GPRC5D drugs.
Multiple myeloma patients in the iMMagine-1 study were less heavily pretreated than in the CARTITUDE-1 trial of Carvykti, but Gilead noted that more patients receiving anito-cel had high-risk features.
The Swiss drug maker presented Phase II data at ASH for rapcabtagene autoleucel, also called YTB323, showing a 65% CR rate in relapsed/refractory diffuse large B-cell lymphoma.
The biotech’s Phase III data with an oncolytic immunotherapy for non-muscle invasive bladder cancer shows high complete response rates, duration of response and good safety/tolerability.
Ahead of ASH 2024, Ichnos Glenmark Innovation’s CEO outlines to Scrip the promising profile of the alliance’s early stage trispecific versus existing bispecific antibodies in relapsed/refractory multiple myeloma, including a significant overall response rate.
Both companies are seeking to license their products as they try to stretch their dwindling cash.
The Swiss pharma acquired Kate Therapeutics for up to $1.1bn, gaining preclinical gene therapies for neuromuscular conditions and novel platform technology.
The company is trading below cash after admitting that an adverse event in a Rett syndrome trial left a patient critically ill.
Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.
The biotech anticipates treating 74 patients with its gene therapies this year, with 30 more already scheduled for 2025.
Analysts also looked into how much differentiation there was between data for the gene therapy NGN-401 and what Taysha has shown with TSHA-102 in the same disease.
Aucatzyl will look to take market share from Gilead’s Tecartus, and the US approval is a boost for the firm and the wider UK biotech sector.
The drug maker said in its third quarter earnings report that competition from other therapeutic classes, particularly bispecifics, resulted in a slight decline of sales for the CAR-T.
The otherwise encouraging data were marred by one patient death, but the company attributed it to the busulfan conditioning regimen and not BEAM-101.
Lyell believes ImmPACT’s dual CD19/20-targeted CAR-T could offer better response rates than Yescarta or Breyanzi and will focus on IMPT-314 while terminating much of its own pipeline.
The gene-editing therapy produced functional cures in eight of 11 patients receiving the go-forward 50mg dose, but the rate fell short of what some investors had hoped for.
Multiple Japanese ventures presented updates on their novel pipeline assets and modalities at BioJapan, including cell and gene therapies and combination treatments, despite lingering financing challenges.
Ocuphire will shift toward gene therapy for inherited retinal degeneration via a merger that will take Opus Genetics public. The firm will also be responsible for one of Ocuphire’s pipeline projects.