Vertex Pharmaceuticals is halting work on an experimental drug for a rare protein deficiency after safety problems were reported in Phase 2 testing.
The study was evaluating the small molecule, VX-814,... Read more »
Codiak BioSciences now has $82.5 million to fund clinical tests of a new class of medicines based on exosomes, tiny bubbles formed by cells that the company harnesses to carry therapeutic... Read more »
Roche made a splash with its acquisition of Spark Therapeutics, whose Luxturna, a gene therapy for an inherited form of vision loss, was the first such therapy to win FDA approval.... Read more »
Gossamer Bio’s lead drug, an experimental treatment for asthma and allergies, has failed in two mid-stage clinical trials but the biotech sees enough in the data to pursue further development in... Read more »
SQZ Biotechnologies, which already has an alliance with Roche focused on developing new cancer cell therapies, is now looking to sell shares to the public to finance its research of potential... Read more »
An opioid that Avenue Therapeutics reformulated for treating post-surgical pain in hospital settings has been rejected by the FDA due to safety concerns.
The Avenue (NASDAQ: ATXI) drug is an... Read more »
An Alkermes antipsychotic treatment designed to offer the benefits of a commonly prescribed medicine, but without the weight gain side effect, won the backing of an FDA advisory committee on Friday.... Read more »
It’s October: a time of days growing shorter, leaves changing color, and phone calls in the wee hours that rouse scientists from slumber notifying them they’ve won the highest honor in... Read more »
Arrowhead Pharmaceuticals’ gene silencing treatment for a rare liver disease will move through the regulatory process and potential commercialization with the muscle of Takeda Pharmaceutical behind it.
Takeda (NYSE: TAK)... Read more »
The first generation of CRISPR-based therapies showed how the technology can edit DNA, making genetic fixes to unwanted mutations. But those treatments use the CRISPR system as scientists found it in... Read more »
Scientists at microbiome therapy developers often talk about the importance of community, how the bugs in our gut act in concert in addition to their individual roles. That’s important for microbiome health... Read more »
Bristol Myers Squibb is bolstering its cardiovascular drug lineup with a $13.1 billion deal to acquire MyoKardia, a company whose experimental therapy for a rare type of heart disease is on... Read more »
Amid the bombast of Tuesday’s presidential debate, viewers caught some discussion about the response to the coronavirus pandemic and efforts to develop a COVID-19 vaccine. In the crosstalk, the president made... Read more »
Solid Biosciences has answered the FDA’s outstanding questions about its Duchenne muscular gene therapy, clearing a clinical trial to now resume after patient complications last year led the agency to halt... Read more »
[Updated 10/1/2020. See below.] Genfit is halting all work on its lead drug in the fatty liver disorder nonalcoholic steatohepatitis (NASH) and implementing a corporate restructuring that will cut... Read more »
CStone Pharmaceuticals has touted its experimental drug sugemalimab as potentially the best in its class of cancer immunotherapies. Pfizer is placing a bet on that promise by making a $200 million... Read more »
Ovid Therapeutics and Takeda Pharmaceutical joined forces three years ago to develop a small molecule into a potential epilepsy drug. That research is starting to pay off as the companies now... Read more »
Orphazyme has fresh cash from the sale of shares in the US and Europe, which will support its lead rare disease drug through regulatory review and, if approved, a launch. But... Read more »
Praxis Precision Medicines kept mostly quiet about its research on central nervous system disorder (CNS) drugs until May, when it emerged from stealth and revealed $100 million in cumulative financing.... Read more »
Had things turned out differently, scientists at Galecto might be watching their idiopathic pulmonary fibrosis drug candidate develop in the hands of a big pharmaceutical company. But Bristol Myers Squibb passed... Read more »