Researchers at the Harvard Medical School have developed a universal gene therapy for Diamond-Blackfan Anemia (DBA), a rare genetic disorder characterized by impaired erythropoiesis caused by ribosomal protein mutations. According to a Cell Stem Cell research article, the approach entails controlling the expression of GATA1, a crucial transcription factor in…

PTC Therapeutics’ gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency deficiency has received FDA approval. It is the first-ever gene therapy approved in the U.S. that is directly administered to the brain. The treatment will be marketed under the name KEBILIDI™ (eladocagene exuparvovec-tneq). It is indicated for children and…

In the fall of 2010, I began my doctoral studies at the University of California, San Diego (UCSD). I quickly began to use a model that was novel to me and the world: human pluripotent stem cells (hPSCs) derived cardiomyocytes. When I first thawed them, I was surprised by the…

A Phase I/II study of Lexeo Therapeutics’ gene therapy to treat patients with Alzheimer’s associated with the APOE4 genetic variant has achieved good interim results according to the company. If approved, this therapy (currently known as LX1001) would be ground-breaking as the first gene therapy for Alzheimer’s disease in a…

Children diagnosed with Canavan disease, who have always faced a grim prognosis—the inability to speak, walk, or maintain head control for their short lifespans with this fatal rare neurodegenerative disorder—may soon be treatable, even “curable.” At the 31st annual European Society for Cell and Gene Therapy (ESCGT) meeting, BridgeBio presented…

Without a doubt, tens of thousands of people afflicted with blood cancers—leukemia, lymphoma, multiple myeloma—have had incredibly positive outcomes with CAR T cell therapy. When Emily Whitehead was treated in April 2012 at the age of six, the hope was that she would live another day; if she survived until…

Gene therapy with elivaldogene autotemcel (BlueBird’s Skysona/eli-cel) has been a miracle for children with the rare disease cerebral adrenoleukodystrophy (CALD). Unfortunately, the treatment appears to have caused blood cancer in some patients. Now researchers from Massachusetts General Hospital, BlueBird, and their colleagues are sharing new data on this phenomenon. Skysona…

MeiraGTx has announced that its gene therapy (AAV-GAD) for Parkinson’s disease has passed a midphase trial and they are in discussion with regulators about Phase III. A small study (MGT-GAD-025) of patients receiving the highest dose of the drug showed significant improvement in their Parkinson’s Disease Questionnaire (PDQ-39) score at…

Last year’s Cell and Gene Meeting on the Mesa took place at a lush resort in northern San Diego. The coastal October air was cool and crisp, and the attendees were filled with cozy optimism, clinking glasses and shmoozing with a backdrop of a spectral Southern California sunset. They were…

A gene therapy designed to treat Bothnia dystrophy, a rare form of inherited retinal disease, was well tolerated and improved vision in a Phase I/II clinical trial led by the Karolinska Institute in Sweden. Bothnia dystrophy is an autosomal recessive form of retinal dystrophy that is most common in the…

Pfizer saw positive topline results of its investigational gene therapy for adults with hemophilia A in a Phase III study (AFFINE). The therapy, giroctocogene fitelparvovec, reduced patients’ bleeds for at least 15 months and was generally well tolerated. It comprises a bio-engineered AAV6 capsid and a modified B-domain deleted human…

A new gene therapy treatment for Duchenne muscular dystrophy show promise of arresting the decline of the muscles of those affected by this inherited genetic disease, and perhaps, in the future, repairing those muscles. The laboratory results not only show promise to treat patients with this severely debilitating and incurable…

William Chou, MD, strives to bring more hope to patients with neurodegenerative diseases. And Passage Bio, where Chou is president and CEO, may now have the green light to test their lead gene therapy asset more broadly than the initial focus of frontotemporal dementia (FTD) caused by mutations in one…

Lexeo Therapeutics this week announced positive interim data from early trials of its gene therapy LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. LX2006 was well tolerated with no treatment-related serious adverse events, and provided clinically meaningful improvements in cardiac biomarkers were observed with increasing improvement over time.  FA…

Updated interim data from a Phase I/II trial of uniQure’s “one shot” gene therapy in Huntington’s disease were promising, suggesting AMT-130 is effective and safe. Huntington’s is one of the most common rare diseases. It is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene…