After spending over seven years at Sarepta Therapeutics working towards curing Duchenne muscular dystrophy (DMD) and other rare diseases, Bo Cumbo took the helm at a startup called AavantiBio to try to do something never done before—to target both the brain and the heart simultaneously with gene therapy. Now, this…

Gene transfer for kidney disease is challenging. To address this, a team used “context-dependent selection of AAV capsids.” They found that local delivery of AAV-KP1, but not AAV9, via the renal vein or pelvis effectively transduces proximal tubules with minimal off-target liver transduction. Meanwhile, systemic AAV9, but not AAV-KP1, enhances…

Mitochondria play a crucial role as the powerhouses of cells, generating the energy necessary for cellular functions. For years, they have been seen as a promising target for anti-cancer therapies, yet their impermeable inner membrane has posed significant challenges. Now, Ohio State University scientists combined methods to administer gene therapy…

Gene therapies received back-to-back sickle cell disease (SCD) approvals in December 2023, but uptake has been slow largely because of cost: Bluebird bio’s Lyfgenia costs $3.1 million, and Vertex’s and CRISPR Therapeutics’ partnered Casgevy has a list price of $2.2 million. Both gene therapies are one-time infusions meant to cure…

REGENXBIO has already dosed the first patient in the next phase of the pivotal trial of its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD). Based on positive Phase I/II results, this week the company received FDA approval for expanding to a Phase I/II/III. They also received access to accelerated…

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…