Deaf toddler can hear after world-first gene #therapy
A #British toddler who was born deaf has had her hearing restored by groundbreaking gene therapy. #Researchers say it marks a new era in the #treatment of #deafness.
While there are numerous caveats, there are now two gene editing therapies available for people with Sickle Cell Disease, and early therapies have been very successful. It's nowhere near cheap at over $2M for one and over $3M for the other, but it's a start I suppose. Only the 1% can afford such therapy for now, but if sanity ever returns to society, I would hope that empathy will eventually win out over profits in the end. #science#crispr#genetherapy#sicklecelldiseasehttps://youtu.be/uHWD8RSw4As
Deaf since birth, an 11-year-old boy now hears thanks to gene therapy that replaced a faulty gene with a healthy one, offering hope for millions with similar hearing loss.
In a medical milestone, Seattle biotech Immusoft used genetically modified B cells from a patient to fight disease, marking the first test of engineered B cells in humans.
Today the FDA approved 2 gene therapies to treat sickle cell disease, including the first CRISPR-based therapy.
Bluebird Bio, who has the ~$1M more expensive and not-CRISPR therapy, has a single card left to play in this competition: It's been conducting a year-long dress rehearsal for this moment
The UK authorizes world-first gene therapy for sickle cell disease and B-thalassemia.
How’s it work? Scientists use CRISPR to edit the faulty gene in a patient’s stem cells in the lab so that they make functioning hemoglobin. Then, they infuse the cells back into the patient.
Results? Recall many sickle cell patients have frequent pain crises and anemia. And B-thal pts can need blood transfusions every month.)
-The trials are ongoing, but of eligible sickle cell 1/2
Antibody/Gene Therapy Biotech Gains $138 Million in Early Funds
A developer of synthetic antibodies for neurodegenerative disorders delivered like gene therapies is raising €129 million ($US 138 million) in its first venture funding round.
The Food and Drug Administration is gearing up to review what could be the first approved gene therapies for sickle cell disease — treatments that could offer functional cures and transform what it means to have this diagnosis.
This is tremendously exciting. At the same time, there are real concerns about affordability and access to what is a risky and aggressive treatment
A new company spun-off from a university lab consortium is advancing gene therapies delivered through the lungs to treat rare inherited respiratory diseases.
Regeneron Pharmaceuticals, a maker of synthetic biologic treatments, is acquiring Decibel Therapeutics, developer of gene therapies to treat hearing loss and balance disorders.