BioCentury | Dec 31, 2020
Emerging Company Profile

Cardior: targeting microRNAs to treat heart failure

Cardior plans to build on early clinical data for its antisense oligonucleotide in heart failure patients by moving the RNA therapy into niche indications where it believes the product can help dysfunctional...
BioCentury | Dec 4, 2020
Distillery Therapeutics

Scripps team discovers RNA-targeting small molecule for myotonic and corneal dystrophies

DISEASE CATEGORY: Musculoskeletal; ophthalmic disease INDICATION: Myotonic dystrophy type 1 (DM1); ophthalmic A Scripps Research team led by Expansion Therapeutics Inc. co-founder Matthew Disney has designed an RNA-targeting small molecule that could treat DM1 and Fuchs...
BioCentury | Nov 14, 2020
Product Development

ASH 2020 snapshot: efficacy data in hot emerging areas

This year’s ASH meeting provides an opportunity to take stock of clinical progress in hot target, modality and disease areas: CD47 inhibitors, allogeneic CAR T cells and sickle cell disease. Abstracts...
BioCentury | Nov 13, 2020
Product Development

Next-generation sickle cell readouts at ASH: Data Byte

The move toward sickle cell disease therapies that address the pathology’s root causes is reflected in promising early-stage readouts at ASH, which include both disease event rates and changes in red blood cell biology. The...
BioCentury | Aug 28, 2020
Distillery Therapeutics

Exon skipping therapy for Batten disease

DISEASE CATEGORY: Neurology INDICATION: Neurology Antisense oligonucleotides that induce exon skipping in lysosomal membrane protein CLN3 could treat CLN3 Batten disease, a neurodegenerative lysosomal...
BioCentury | Aug 25, 2020

Seeing path for cholesterol-lowering program, Forbion launches start-up to house former Dezima molecule

Five years after reaping a strong return from cholesterol-lowering molecule obicetrapib, Forbion is again investing in the program based on a belief it can serve some dyslipidemia patients where others in its class fell short....
BioCentury | Jul 25, 2020
Product Development

NIH, HHS fast-tracking point-of-care, pooled tech as way out of testing crunch

The U.S. government is taking non-conventional paths to point-of-care and pooled technologies in its push to bulk up and speed up the country’s strained COVID-19 testing capacity. In HHS’s case, the department is scrambling to...
BioCentury | Jul 3, 2020

On heels of $100M raise, Annexon joins burgeoning IPO queue

Two days after raising $100 million in a series D round heavy with crossover investors, Annexon has filed to go public on NASDAQ. The filing adds to a burgeoning IPO queue and caps another busy...
BioCentury | Jun 27, 2020
Product Development

Modeling harmony: learning from preclinical coordination of COVID-19 countermeasures

The all-hands-on-deck COVID-19 response provides a glimpse of what’s possible when drug developers converge around solving a problem. One of the most important developments might be in preclinical research where consensus on harmonized models and...
BioCentury | Jun 10, 2020
Emerging Company Profile

Autobahn: fine-tuning thyroid agonism to remyelinate axons

Autobahn, which launched with a $76 million series B Tuesday, joins at least six other companies with disclosed remyelination programs for multiple sclerosis. But the biotech thinks its prodrug, slated to enter the clinic next...
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