Disease Science Virus 
Broad Institute
JULY 24, 2025
Murphy New treatments are sorely needed for Ebola virus infections, which cause rare, yet severe, outbreaks. Related news Imaging combined with genetic screening of cells enhances genomic discoveries Although outbreaks of Ebola virus are rare, the disease is severe and often fatal, with few treatment options.
Broad Institute
MAY 21, 2025
Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. The tools are known as enhancer AAV vectors.
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Drug Target Review
JULY 21, 2025
Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. Viral vectors use a virus’s blueprint to deliver genetic material into cells.
The Pharma Data
JUNE 24, 2025
Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers. To date, more than 160 monoclonal antibody therapies targeting nearly 100 disease-related proteins have received regulatory approval globally.
Broad Institute
JULY 21, 2025
There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& & The new study, published in Cell , is the first time prime editing has been used to treat a neurological disease in animals, offering hope for treating people with AHC and other genetic brain disorders.
The Pharma Data
JUNE 13, 2025
Importantly, this will enable them to produce mRNA vaccines during periods between health crises, retaining trained personnel, technical expertise, and production capacity while retaining the ability to respond quickly in the event of future disease outbreaks.
Codon
JUNE 5, 2025
Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.
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