BioSpace18h
Arbor Raises $73.5M To Ride Out Gene Editing’s ‘Gartner Hype Cycle’
The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.
This Founder’s Gene Therapy Company Helped Blind Kids See Again
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
Fierce Biotech18h
Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. | As other gene editing programs ...
Using machine learning tools to detect gene mutations from leukemia cell images
Gene mutations in acute myeloid leukemia (AML) cells can guide treatment options, and machine learning can rapidly guess the ...
BioPharma Dive8d
3 ways to accelerate development and de-risk cell and gene therapy manufacturing
Development strategies designed to reduce the unknowns so that cell and gene therapy innovators can develop robust processes ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the “severity” of the patient’s case had not previously been seen with the therapy, ...
Fierce Biotech8d
Beam gene therapy for protein deficiency passes early safety test, sets efficacy bar in space: analyst
The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), ...
Mutated DNA Restored to Normal in Gene Therapy Advance
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
CNN29d
What a $2 million per dose gene therapy reveals about drug pricing
Because his drug was a gene therapy, public grant money and private ... The CEO, John Carbona, then 54, had run medical device and equipment companies, but he had no background in drug development.
Preclinical study demonstrates improved gene therapy approach to treating a rare and devastating disease in children
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Science Daily7d
Gene Therapy News
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...