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Faulty gene likely to be cause of severe learning difficulties, study finds
A faulty gene is likely to be responsible ... “But it also comes with some benefits potentially in the future, because it means that it will be potentially more feasible to develop a therapy that ...
New Atlas1d
RNA therapy an effective alternative to lifelong, painful eye injections
An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...
Science News1d
A common drug may help treat a rare genetic disease
Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...
Science Daily1d
New take on immunotherapy reinvigorates T cells by blocking uptake of energy-sapping cancer byproducts
Blocking a protein that imports lactic acid, a metabolic byproduct of cancer cells, reinvigorated exhausted T cells and led to improved tumor control in mouse models of cancer, according to a new ...
Science Daily1d
Groundbreaking research unlocks precision therapy for drug-resistant leukaemia
Scientists investigated how an inherited genetic variation common among East Asians contributes to drug resistance in cancer cells, driving more aggressive cancer growth. The team trialled a precision ...
Ocugen (OCGN) Q3 2024 Earnings Call Transcript
Ocugen ( OCGN -6.48%) Q3 2024 Earnings Call Nov 08, 2024, 8:30 a.m. ET Good morning and welcome to Ocugen's third quarter 2024 financial results and business update. [Operator instructions] I will now ...
TMCnet2d
Ocugen Provides Business Update with Third Quarter 2024 Financial Results
While gene therapy remains the primary focus for the Company ... including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data ...
sicklecellanemianews2d
BEAM-101 gene-editing stem cell therapy shows promise in SCD
BEAM-101, a gene-editing stem cell therapy, showed early signs of efficacy among the first treated sickle cell patients in a ...
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...
labiotech3d
Cystic fibrosis: Five biotechs breathing life into the therapeutic space
In this article, we take a look at five biotech companies developing potentially transformative medicines to treat cystic ...
Can a combo of “active” therapies help lower dementia risk?
A combination of two “active” therapies may help slow cognitive decline in older adults, especially those with a type of ...
Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic ...