BioCentury | Jun 30, 2020
Product Development

Vertex, NHS in deal to make CF triplet available in England once EC approves

It took four years for Vertex to reach an agreement with England’s NHS for access to its first combination cystic fibrosis therapy. But there won’t be any wait for the biotech’s highly anticipated triple therapy...
BioCentury | Apr 27, 2020

Vertex continues its march into neuromuscular diseases with Affinia deal

Vertex is diving deeper into genetic therapies for neuromuscular diseases through an $80 million deal with Affinia to develop new AAV vectors for the indications. Affinia Therapeutics will receive $80 million in up front and...
BioCentury | Apr 15, 2020
Emerging Company Profile

Backed by Third Rock-led syndicate, MOMA seeks new ATP-powered targets by interrogating 'molecular machines'

Third Rock Ventures led a syndicate that invested $86 million in a series A round for MOMA Therapeutics, a newly launched start-up that will seek to interrogate a class of "molecular machines" that drive cellular...
BioCentury | Apr 4, 2020

Investors are seeking only one thing: a sign the pandemic tide is shifting

The usual clinical and commercial catalysts are taking a back seat in the second quarter as buysiders are almost solely focused on COVID-19 milestones that will stabilize the market. On the other hand, the virtual...
BioCentury | Apr 3, 2020
Distillery Therapeutics

IRE1 activation to treat cystic fibrosis and Pendred syndrome

DISEASE CATEGORY: Pulmonary; otologic INDICATION: Cystic fibrosis (CF); hearing loss Activating IRE1 could treat diseases caused by defective trafficking of membrane proteins to the cell surface, including CF and Pendred syndrome. Pendred syndrome is an...
BioCentury | Mar 26, 2020
Emerging Company Profile

ReCode: merging RNA therapies and organ-specific delivery for respiratory diseases

ReCode Therapeutics launched on Thursday with an OrbiMed- and Colt Ventures-led $80 million series A round to pair RNA therapeutics for rare respiratory diseases with an organ-specific lipid nanoparticle delivery technology. The company was formed...
BioCentury | Jan 31, 2020
Distillery Therapeutics

CRISPR correction of CFTR mutations to generate autologous transplants to treat cystic fibrosis

DISEASE CATEGORY: Pulmonary INDICATION: Cystic fibrosis (CF) Autologous transplants for CF patients generated from patient-derived upper-airway basal stem cells edited via CRISPR-Cas9 could treat the genetic disease. The transplants were created by: culturing 3-D organoids...
BC Extra | Jan 16, 2020
Company News

Galapagos to build fibrosis pipeline via extended Fibrocor partnership

In a move that could refill its fibrosis pipeline, Galapagos expanded its year-old collaboration with Fibrocor, adding options to exclusively license four fibrosis programs. Under the original deal, announced December 2018, Toronto-based Fibrocor Therapeutics L.P...
BioCentury | Jan 1, 2020

Fewer FDA approvals in 2019, but a basket of firsts

While the year held fewer approvals of new molecular entities than 2018, it was not short of major achievements. FDA greenlit several drugs with innovative modalities, against novel targets and for diseases that have long...
BioCentury | Nov 1, 2019
Product Development

Vertex’s triple therapy will reshape the landscape of cystic fibrosis trials

FDA approval of Vertex’s long-awaited triple therapy for cystic fibrosis is likely to seal the company’s dominance in the indication for years to come, with competitive threats barely touching the horizon. Moreover, uptake of the...
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