Advanced Therapies
The Pierre Fabre-partnered allogeneic T-cell immunotherapy is already available in Europe but hopes of a US approval have been hit by a complete response letter citing problems at a third-party manufacturing facility.
After ditching its clinical-stage ex vivo candidate in December, the CRISPR-based company is starting all over again with a focus on in vivo therapies and upregulating beneficial genes.
More than 40 industry executives shared their views on where the biopharma industry stands as it enters the new year. Innovation remains the sector’s driving force, but after two years of capital constraint, due diligence is key.
From gene therapies to small molecule drugs, investors seemed to have eschewed the risks of biotech company investment propositions in 2024 but last year’s disappointments provided good lessons going forward.
Following promising Phase II data presented at ASH on the company's allogenic leukemia-derived dendritic cell vaccine, Mendus has secured support from the FDA and the EMA for pivotal studies starting later this year.
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
The cell therapy won approval for pediatric steroid-refractory acute graft versus host disease after two complete response letters over the last four years.
The struggling Japanese pharma group is aiming to grow its new cell therapy business in the US and Japan to $2.3bn by the late 2030s.
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.