New UAB-developed gene therapy for ALS receives orphan drug designation
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...
Tag: gene
Eating to Downregulate a Gene for Metastatic Cancer
Women with breast cancer should include the “liberal culinary use of cruciferous vegetables.” Both the Women’s Intervention Nutrition Study and ...
Sarepta Stops Stop Shipments of Muscular Dystrophy Gene Therapy After Second Patient Death
A patient has died after receiving Sarepta Therapeutics’ approved gene therapy for Duchenne muscular dystrophy — the second fatality reported ...
Second patient dies after getting Sarepta’s Duchenne gene therapy
Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at drewqjoseph.45.Sarepta Therapeutics ...
We’re reading about Roche antibiotic, a gene therapy patient death
Good morning, everyone, and welcome to another working week. We hope the weekend respite — which was longer than usual ...
What Baby KJ means for future of CRISPR gene editing industry
For the ailing gene editing industry, hope came earlier this month in the tiny, smiling, fuzzy-headed form of KJ Muldoon. ...
Single gene found to play a big role in how the liver stores energy
A new study published in Science Advances reveals that a single gene plays a big role in how the liver ...
Gene Therapy Biotech MeiraGTx Strikes Deal Bringing Generative AI to Pivotal Parkinson’s Trial
MeiraGTx has one gene therapy in a potentially pivotal clinical trial for cancer patients and another gene therapy ready to ...
Gene Therapy in an Implant: Neurotech Lands First FDA Approval in Rare Vision Disorder
Protein therapies for the eye must be dosed as frequent injections. Gene therapy offers one-time treatment, but must also be ...
Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity
Bluebird Bio, a company that steered three gene therapies to FDA approval but struggled to commercialize them, has found the ...
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