Advanced Therapies
Tessera, which claims its gene-writing technology offers functionality beyond gene-editing, hopes Regeneron can accelerate development of its preclinical alpha-1 antitrypsin deficiency drug.
With Gilead reporting that both of its approved CAR-Ts saw sales declines as other products saw growth, a mixture of different competitive headwinds seems to be behind the disparities.
The CEOs of two early-stage biotechs explain to Scrip why investor interest in ocular gene therapies is surging and how they aim to attract big pharma partners.
The biotech will present data at the upcoming ASH meeting on three patients who achieved PRs or VGPRs with minimal residual disease negativity and no cases of neurotoxicity.
US FDA approval of the intrathecal formulation of Zolgensma means many more patients with spinal muscular atrophy will be eligible for the gene therapy.
The company announced data from patients enrolled after a protocol amendment designed to bring the study in line with non-muscle invasive bladder cancer (NMIBC) standards of care.
Deal Snapshot: Eli Lilly has become increasingly interested in genetic treatments for the eye and has secured the rights to a high-profile gene therapy for a rare form of childhood blindness.
The company’s third quarter earnings fell by $67.8m year-over-year, reflecting the tumultuous situation around its DMD therapy Elevidys in June.
UniQure’s plan for a 2026 BLA filing of gene therapy AMT-130 for Huntington’s is uncertain, as the FDA apparently reversed course after indicating it would consider accelerated approval.
The company said in its third quarter earnings that it would focus on areas that better foster growth for the company as the hemophilia A treatment only brought in $3m in Q3.
The company reported that a patient went to the emergency room after experiencing grade 4 transaminase elevations, which unlike a similar event in May caused symptoms.
The Basel-based company has faith in its existing rare disease commercial platform to successfully market the newly-acquired assets.
The drugmaker has made numerous moves in the field this year, with the latest deal bringing in ixo-vec, a Phase III candidate for wet AMD.
Data presented at the Berlin meeting highlight the potential of the company's cell therapy anzu-cel to redefine the treatment paradigm for uveal melanoma.
Positive data from the PSMAddition trial potentially doubles the number of prostate cancer patients eligible for treatment with the Swiss major’s big-selling radioligand therapy but a discussant at ESMO was not convinced by the study.
The drugmaker is joining the rush to acquire an in vivo CAR-T therapy developer as concerns mount about whether the health care system can accommodate cell therapies’ dramatic growth.
While some big pharma companies have exited cell and gene therapy, Novartis, Astellas, Gilead’s Kite and AstraZeneca’s Alexion are diving back in, executives said at ARM’s Cell and Gene Meeting on the Mesa.
While there may be the beginnings of a turnaround in biopharma capital markets, gains in the cell and gene therapy space may lag the broader sector recovery, noted speakers at ARM’s Cell and Gene Meeting on the Mesa.
It remains to be seen whether the Trump administration’s most favored nation drug pricing policy will trickle down from big pharma to small biotech, but executives said at ARM’s Cell and Gene Meeting on the Mesa that they are preparing strategies now to manage the future.
The companies announced positive Phase II results for AlphaMedix, a lead 212-based alpha emitter therapy for gastroenteropancreatic neuroendocrine tumors.