Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…
According to a story from Chemical & Engineering News, the American Society of Gene & Cell Therapy annual meeting was more crowded than usual this year. In fact every event…
According to an article from ReliaWire, a sizable portion of the global population is likely to have an immunity to the inert viruses scientists use to administer gene therapy in…
According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…
According to a story from andreeger.com, gene therapy, a type of medical treatment in which genes are modified in order to treat disease, has become a hot button topic in…
According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…
Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…
An article recently published by the Guardian shared the stories of three families affected by recent breakthroughs in gene therapy treatments. What is Gene Therapy? Gene therapy is an increasingly…
According to a publication from the Stamford Advocate, the Connecticut-based Alliance for Cancer Gene Therapy (or ACGT) has welcomed a new president and CEO in Kevin Honeycutt. Honeycutt has announced…
When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
Last week the Biotechnology Innovation Organization released a letter to the FDA, commenting on the latter's draft of a document that proposes a new set of guidelines for the treatment…
Two children from a Brooklyn family were born with an ultra rare genetic disease called Canavan disease. Their parents' research revealed that gene therapy could possibly treat the disease. Unfortunately,…
A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
In a Washington Post article by Ben Guarino, new cancer treatments lasso the power of naturally occurring immune cells in the body. Researchers are looking into ways modify them to…
Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…
At six years old, Benny was unable to sit up, roll over and scoot like other babies his age. Missing all the major milestones, his mother Jennie knew that something…
A new gene therapy treatment may be on the horizon for Oculopharyngeal Muscular Dystrophy patients. Find out more below, or keep up with the original story at musculardystrophynews.com. Oculopharyngeal Muscular…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
As the world turns, so do advancements in gene therapy. Technology is changing our world so drastically that soon gene therapy will be simplified into a one-shot treatment. With it,…
Sickle cell anemia results from a single mutation in the hemoglobin gene. The mutation creates a protein which has an atypical structure. This altered structure throws a wrench in the…
In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent. A recent press release issued by…
Little Emily Whitehead is looking attentively at a butterfly that has landed on her finger. She coos at it and lets it rest on the tip of her thumb peacefully.…
Pharma giant Novartis's new cancer drug tisagenlecleucel passed a critical test by winning the support of a federal advisory panel - setting it up for final FDA approval in September.…
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