Gene 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.
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.
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 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.
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.
Speakers at ARM’s Cell and Gene Meeting on the Mesa highlighted challenges of CAR-T’s evolution from niche to mainstream therapy given health care providers’ limited capacity to deliver the complex care associated with the products.
FDA’s former CBER director is joining industry as senior VP of molecule discovery and head, infectious disease at Eli Lilly.
Chiesi and the Boston-area biotech will co-develop a CRISPR-Cas gene-editing therapy for primary hyperoxaluria type 1. They hope to provide a curative therapy for the rare disease.
The regulator has agreed to a six-month preliminary analysis of its pivotal study which could accelerate filing of TSHA-102 for Rett syndrome.
Emerging company profile: PulseSight Therapeutics is developing non-viral gene therapies for AMD, led by PST-611 for geographic atrophy. Its electro-transfection platform aims for durable protein expression and reduced injection frequency.
The privately owned biotech said it would pause its Phase I/II study of CAP-002 in STXBP1-related disorders after the first patient in the study died.
The company took a prophylactic C3 inhibitor out of the immunomodulatory regimen used in its Phase II study of RP-A501, lowered the dose and adjusted its use of eculizumab.