BioCentury | Nov 25, 2020
Product Development

Data Byte: a snapshot of the clinical cutaneous T cell lymphoma pipeline

At least 14 therapies are in the clinic to treat cutaneous T cell lymphoma, a rare cancer of T lymphocytes that primarily affects the skin. The most common subtypes of CTCL are mycosis fungoides and...
BioCentury | Oct 24, 2020
Translation in Brief

UNC Chapel Hill Angelman gene therapy rescues motor function; plus data backing planned Pacylex, Kronos clinical trials and more

AAV gene editing therapy for Angelman syndromeUniversity of North Carolina at Chapel Hill scientists have created a CRISPR gene therapy for Angelman syndrome that ameliorated motor function deficits in a mouse model of the maternally...
BioCentury | Sep 5, 2020
Translation in Brief

Matt Disney’s microRNA-targeting angiogenesis compound; Arrakis’ platform to find compounds’ RNA targets; dual target CAR Ts for HIV and more

Scripps microRNA-targeted VEGF-A stimulatorA Scripps Research Institute team led by Matthew Disney has designed a compound that targets a  precursor of miR-377, a direct inhibitor of angiogenesis...
BioCentury | May 28, 2020
Distillery Therapeutics

microRNA cluster identified as targets for ischemic stroke

DISEASE CATEGORY: Neurology INDICATION: Stroke Targeting the miR-15a/miR-16-1 cluster to enhance blood-brain barrier function could treat ischemic stroke. In mice with middle cerebral artery occlusion-induced stroke, endothelial cell-specific knockout of the miR-15a/miR-16-1 cluster reduced infarct...
BioCentury | May 20, 2020
Product Development

BARDA takes steps to secure supply chain as more companies look to scale up manufacturing

HHS is taking steps to shore up the U.S.’s national supply chain and manufacturing capabilities after global disruptions in the early days of the COVID-19 crisis. The agency’s BARDA has closed a four-year, $354 million...
BioCentury | May 15, 2020
Product Development

Repurposed autoimmune therapies head for controlled COVID-19 studies in Europe

Abivax and Immunic are launching COVID-19 trials of their autoimmune therapies in France and Germany, respectively, meeting regulators’ demands for large, controlled studies to demonstrate therapeutic efficacy. In the U.S., the patent office is fast-tracking...
BioCentury | Feb 28, 2020
Distillery Therapeutics

An antisense oligo inhibitor of miR-132 for heart failure

DISEASE CATEGORY: Cardiovascular INDICATION: Heart failure The miR-132 inhibitor CDR132L could treat heart failure. In pigs with post-myocardial infarction heart failure, CDR132L improved cardiac function, prevented end-systolic volume enlargement and reduced histologic evidence of interstitial...
BioCentury | Feb 1, 2020
Preclinical News

Cardior eyes heart failure cure with miRNA inhibitor

Cardior unveiled data on Friday that demonstrates its first clinical candidate goes beyond treating the cardiovascular symptoms of heart failure to reversing established disease in animal models. Cardior Pharmaceuticals GmbH, which spun out of Hannover...
BioCentury | Jan 21, 2020
Tools & Techniques

Disney lab uncovers another RNA-binding small molecule for triple-negative breast cancer

Matthew Disney is attacking triple-negative breast cancer again with the latest iteration of his RNA-binding small molecule technology, which marries the concept with targeted RNA degradation to eliminate disease-causing RNA segments. In a paper published...
BioCentury | Dec 20, 2019

CAPN3 gene therapy for limb-girdle muscular dystrophy type 2A

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular dystrophy An adeno-associated viral vector deliverying CAPN3, a calcium-dependent protease expressed at low levels in the heart and primarily expressed in skeletal muscle, could treat limb-girdle muscular dystrophy type 2A...
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