A decentralized clinical trials model is especially important for patients with rare, genetic, or complex diseases. Learn how this model increases access to trials, bringing new therapies to rare disease patients faster.
Clinical drug development is moving away from traditional approaches... Read more »
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, as a treatment for alpha-1 antitrypsin deficiency, which can lead... 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 cargo.
Late Tuesday, Codiak priced its offering of 5.5 million... 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. Now the Swiss pharmaceutical giant is looking to bolster its... 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 new treatments for both cancer and infectious disease.
In paperwork... 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. But even some of the panel members who supported the... 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 their fields.
This week, the 2020 Nobel Prize in physiology... Read more »
A US Food and Drug Administration advisory committee meeting on Biogen, Inc.’s Alzheimer’s drug aducanumab will offer some insight into the amount of flexibility the agency and its external experts are willing to exercise for neurodegenerative diseases with high unmet need,... 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) has agreed to pay $300 million up front in a... Read more »
Science is the way out of this pandemic—and scientific breakthroughs are on the ballot on November 3. To help you get ready for Election Day, BIO’s initiative Biotech Votes has resources to help you get registered and get informed.
First, make... 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 nature, which means it still has limitations when applied to... 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 and for microbe-based therapies, says Emily Drabant Conley, CEO of... 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 an unsupported claim that we are weeks away from having... Read more »