BioCentury | Nov 10, 2020
Product Development

First biomarker data from pioneering tau program in Alzheimer’s cloud use of antibodies for the target

Data presented at the annual CTAD meeting hint that anti-tau therapy semorinemab from Genentech and AC Immune may have engaged its target yet failed to decrease tau aggregation in a Phase II study to treat...
BioCentury | Nov 5, 2020
Product Development

FDA signals relatively clear path ahead for Alzheimer’s therapy aducanumab

FDA appears to be leaning toward approval of the first theoretically disease-modifying therapy for Alzheimer’s disease, or any neurodegenerative disorder, based on two incomplete Phase III trials, one...
BioCentury | Oct 3, 2020
Translation in Brief

A SARS-CoV-2 genome degrader; neutralizing influenza B mAbs; Fulcrum’s sickle cell therapy increases fetal hemoglobin and more

Matt Disney’s small molecule RNA degraders for COVID-19A Scripps Research team led by Expansion Therapeutics Inc. founder Matthew Disney published an ACS Central Science study describing a small molecule that binds a SARS-CoV-2 hairpin RNA structure that...
BioCentury | Sep 29, 2020
Distillery Therapeutics

Small molecule kinase inhibitor for cystic fibrosis with splicing mutation

DISEASE CATEGORY: Pulmonary INDICATION: Cystic fibrosis (CF) A small molecule inhibitor of four kinases that regulate splicing could treat CF caused by the most common disease-associated CFTR splicing...
BioCentury | Sep 29, 2020
Product Development

Alzheimer milestones & vaccine politics: a BioCentury podcast

Alzheimer’s disease therapies continue to tease and torment biotech investors. In the latest BioCentury This Week podcast, BioCentury editors discuss the road ahead for Biogen’s closely watched therapy aducanumab and anti-tau therapies, whose most significant...
BioCentury | Sep 24, 2020
Product Development

The road ahead for the tau hypothesis of Alzheimer’s disease

The Phase II miss of the first tau antibody to reach that stage continue the tradition in Alzheimer’s disease of bad news with hard-to-interpret ramifications for the target. At a minimum, the data call into...
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 | Aug 28, 2020
Distillery Therapeutics

CRISPR-based gene editing for Wiskott-Aldrich syndrome

DISEASE CATEGORY: Autoimmune INDICATION: Autoimmune Gene editing of mutated WASp in hematopoietic stem and progenitor cells (HSPCs) could treat Wiskott-Aldrich syndrome. The therapy uses an adeno-associated virus serotype 6 (AAV6) vector to deliver a CRISPR-Cas9...
BioCentury | Aug 27, 2020
Distillery Therapeutics

Retrotransposons as acute myelogenous leukemia targets

DISEASE CATEGORY: Cancer INDICATION: Acute myelogenous leukemia (AML) Targeting LTR2B and the related LTR2, both long terminal repeat retrotransposons, could treat AML by reducing expression of the lipoprotein component APOCI. Analysis of a The Cancer...
BioCentury | Aug 27, 2020
Distillery Therapeutics

IL-25 to mitigate GvHD in the GI tract after transplant

DISEASE CATEGORY: Transplant INDICATION: Graft-versus-host disease (GvHD) IL-25 could protect against gastrointestinal GvHD by reducing the loss of goblet cells, which contribute to barrier function through mucus and antimicrobial peptide production. In patients who had...
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