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 »
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 the study for a second time.
Cambridge, MA-based Solid Bio... 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 equity investment in CStone for rights to that drug in... 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. Now the company is aiming to raise about that amount... 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 on its option to acquire Galecto leaving the biotech to... Read more »
In biotech, you can go home again. Four years ago, Illumina spun out cancer diagnostics startup Grail. This week, the gene sequencing giant announced it is acquiring Grail in an $8 billion cash and stock deal.
Grail set out with the... Read more »
Many drugs work by binding to a protein and blocking it. Technology for eliminating disease-causing proteins altogether remains experimental, but this approach has gained ground in the past year with hundreds of millions of dollars raised and compounds advanced into clinical... Read more »
Amyotrophic lateral sclerosis (ALS) has no cure and few available treatments but the past decade has revealed new knowledge about the pathology of the disease. Libra Therapeutics is turning that research into potential therapies, and it now has $29 million to... Read more »
The key to Athira Pharma’s approach to Alzheimer’s disease is already inside each of us: a repair mechanism that can regenerate damaged neurons. What’s needed to begin the process is a pharmacological kickstart.
Athira (NASDAQ: ATHA) is developing a drug... Read more »
The 2020 National Xconomy Awards complimentary two-week, 11-part, on-demand Meet the Finalists daily webcast series continues this Tuesday and Wednesday at 11 a.m. ET. Registration is complimentary and includes access to a networking app with already more than 600 life science... Read more »
From Our Editors · Special Report
Becoming the Cell & Gene Therapy Hub
Sponsored · Whitepaper
Explore the different sources of funding, their relative merits, and how you should best utilize that funding at each stage of your research
Brex
Sponsored · Whitepaper
Virtual/hybrid trials and decentralized research can better integrate healthcare into patients’ lives and accelerate approval of new medications
PRA Health Sciences
