A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...

May 15, 2025 — A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant ...

This technique streamlines quality control in gene therapy vector production, potentially accelerating manufacturing and improving clinical outcomes. This summary was automatically generated using ...

Lexeo Therapeutics laid off 15% of its staff last month as part of a wider plan to siphon $20 million from its pipeline to fund the gene therapy biotech’s lead cardiac programs. One of these ...

About Skyline Therapeutics Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel therapeutic solutions for rare and severe diseases ...

It can also not be given to people with detectable antibodies to variant AAV serotype Rh74, the viral vector used to deliver the gene therapy. Both gene therapies are designed to allow patients ...

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for ...

The 11-year-old will be the first to receive an experimental antisense oligonucleotide gene therapy to treat NARS1 symptoms. “With her being the first, she’s going pioneer the way for a lot of ...

Zevaskyn is an autologous cell-based gene therapy that utilizes a patient's skin cells that have been reengineered to produce functional Type VII collagen. Patients with RDEB suffer from wounds ...

Gene delivery is a process by which foreign DNA is transferred to host cells for applications such as genetic research or gene therapy. Gene delivery methods can be mechanical (e.g. microinjection ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...

“We’re excited to share our latest data with the scientific community at ASGCT, which remains the most prestigious venue for showcasing gene therapy innovations,” said Dr. Nicole K. Paulk, PhD, Siren ...