BioPharma Dive18h
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
University of California17h
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
STAT1d
Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
GEN - Genetic Engineering and Biotechnology News1d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
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 Dive10d
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 ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
EurekAlert!2d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
MedCity News2d
Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys
A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.
News-Medical.Net on MSN3d
In the Thymus We Trust: ProTcell Therapy & the Future of Allogeneic Immunotherapy
In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy is being revolutionized.
FiercePharma3d
Sarepta reports one death after patient received DMD gene therapy Elevidys
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...