Kano Therapeutics Inc. announced it has secured $5 million in seed funding, bringing its total funding to date to $7.1 million. The company plans to use the funding to begin internal pipeline development of ex vivo genetic medicines based on kilobase gene insertion, expand its existing therapeutic collaborations to initiate externally driven preclinical programs, and scale its production capacity.
A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.
Sangamo Therapeutics Inc. put pen to paper on a would-be $1.9 billion-plus deal with Genentech, a unit of Roche AG, to develop intravenously administered genomic drugs for neurodegenerative conditions.
Intellia Therapeutics Inc. has received clearance from the U.K. Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of NTLA-3001 for the treatment of α1-antitrypsin deficiency (AATD)-associated lung disease.
Phase II data showing an 11.1-month improvement in overall survival for advanced ovarian cancer patients treated with the IL-12 immunotherapy IMNN-001 drove up shares of Imunon Inc. by 181% July 30. The results “could usher in the first immune-based therapy for ovarian cancer,” said Stacy Lindborg, president and CEO of the Lawrenceville, N.J.-based company.
Arrhythmogenic cardiomyopathy (ACM) is a severe genetic cardiac disorder caused by mutations in some desmosomal genes. The most frequently affected gene in patients with ACM is PKP2, the loss of which provokes desmosomal instability that leads to activation of downstream disease processes ultimately resulting in life-threatening ventricular arrhythmia and heart failure.
The U.S. Department of Health and Human Services’ Office of the Inspector General disclosed an advisory opinion finding Bluebird Bio Inc.’s fertility support program for a gene therapy treatment could run afoul of federal anti-kickback statutes. That follows a similar opinion against Vertex Pharmaceuticals Inc., and its fertility program associated with gene-editing therapy Casgevy (exagamglogene autotemcel). Vertex subsequently filed a lawsuit.
Epigenetic silencing could prevent the production of proteins that cause pathologies. CHARM (coupled histone tail for autoinhibition release of methyltransferase), a DNA methylation-based editor, suppressed transcription of prion proteins in the brains of mice.
As the hunt goes on for a better treatment in wet age-related macular degeneration (AMD), landmark analyses of two batches of phase II gene therapy data billed as positive were disclosed during the American Society of Retina Specialists annual meeting in Stockholm, where 4D Molecular Therapeutics Inc. and Adverum Biotechnologies Inc. offered findings.
The industry is looking, with renewed hope, to the “promise” of messenger RNA (mRNA) therapeutics for a wide range of diseases beyond COVID-19, and not only in vaccine form but also for gene and cell therapies.
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