Gene therapy news

Here are some recent news updates on gene therapy:

Recent Breakthroughs:

  1. Gene therapy for sickle cell disease: In December 2020, the FDA approved the first gene therapy for sickle cell disease, called Zynteglo (betibeglogene autotemcel). The therapy uses a virus to deliver a healthy copy of the HBB gene to patients' bone marrow cells, replacing the mutated gene that causes the disease.
  2. Gene therapy for spinal muscular atrophy: In May 2020, the FDA approved the gene therapy Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA), a genetic disorder that affects muscle strength and movement. The therapy uses a virus to deliver a functional copy of the SMN1 gene to patients' motor neurons.
  3. Gene editing for inherited diseases: In October 2020, scientists announced a breakthrough in gene editing technology, using a new enzyme called CRISPR-Cas13 to edit genes in human cells. This technology has the potential to treat a wide range of inherited diseases.

Recent Clinical Trials:

  1. Gene therapy for inherited blindness: A phase 1 clinical trial is underway to test a gene therapy for inherited blindness caused by mutations in the RPE65 gene. The therapy uses a virus to deliver a healthy copy of the RPE65 gene to patients' retinal cells.
  2. Gene therapy for muscular dystrophy: A phase 2 clinical trial is underway to test a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscle weakness and wasting. The therapy uses a virus to deliver a functional copy of the dystrophin gene to patients' muscle cells.
  3. Gene therapy for Huntington's disease: A phase 1 clinical trial is underway to test a gene therapy for Huntington's disease, a genetic disorder that causes progressive brain degeneration. The therapy uses a virus to deliver a functional copy of the HTT gene to patients' brain cells.

Recent Research:

  1. Gene therapy for cancer: Researchers have made progress in using gene therapy to treat cancer, including the development of CAR-T cell therapy, which uses genetically modified immune cells to target and kill cancer cells.
  2. Gene therapy for genetic disorders: Researchers have identified several genetic disorders that can be treated with gene therapy, including cystic fibrosis, Huntington's disease, and muscular dystrophy.
  3. Gene editing for inherited diseases: Researchers are exploring the use of gene editing technologies, such as CRISPR-Cas9, to treat inherited diseases, including sickle cell disease and muscular dystrophy.

Recent Regulatory Updates:

  1. FDA approval of gene therapies: The FDA has approved several gene therapies in recent years, including Zynteglo (betibeglogene autotemcel) for sickle cell disease and Spinraza (nusinersen) for spinal muscular atrophy.
  2. European Medicines Agency (EMA) approval of gene therapies: The EMA has approved several gene therapies, including Luxturna (voretigene neparvovec) for inherited blindness and Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy.
  3. Gene therapy guidelines: The FDA and EMA have issued guidelines for the development and approval of gene therapies, including guidelines for clinical trials and manufacturing.