after a viral media campaign saved seven-year-old Josh Hardy's life by
Inc. to work with FDA to provide
access to an investigational therapy, new compassionate access policies are
being debated in the halls of Congress, corporate boardrooms and academic think
tanks. So far, the legislative provisions under consideration represent only
incremental changes to the system.
television and Twitter have long since moved on, last week Rep. Michael McCaul
(R-Texas), co-chair of the congressional childhood cancer caucus, told
BioCentury he is drafting compassionate access legislation that he plans to
introduce in 2015.
Congress is gearing up to reconsider compassionate access to drugs, but wholesale reform looks unlikely.
Oncologists and patient advocates want Pediatric Research Equity Act mandates to study new targeted cancer drugs in children.
G1 Therapeutics aims for first-in-class chemoprotectant with CDK4 and CDK6 inhibitor.
Ossianix is developing small shark-derived single domain antibodies that could hit targets tough to access with traditional approaches.
Oricula is developing a small molecule that could prevent hearing loss during aminoglycoside treatment.
A dearth of programs for blood disorder patients with inhibitors will leave a major unmet need until gene therapies come to market.
Adaptimmune's $104M A round to develop T cell receptors. Plus: Hopp's CureVac bet paying off; Avalon-GSK betting on familiarity, speed.
Johnson & Johnson (NYSE:JNJ) is acquiring Alios BioPharma Inc. (South San Francisco, Calif.) for $1.75 billion in cash. The pharma receives Alios' antiviral assets, including AL-8176 to treat respiratory syncytial virus (RSV). In July, the oral...
Although different companies are pursuing antibodies, vaccines or siRNAs to fight Ebola, many researchers believe it is not a race between them but a combination of approaches that will win the day.
Two Canadian venture firms and a government-funded program have launched Canada's first national health science accelerator, Accel-Rx, to reignite local VC interest in funding early stage biotechs.
Researchers have shown that inhibiting ALK7 can increase energy expenditure in fat cells and suggest the protein could be a target for obesity. Acceleron Pharma has licensed the findings and is pursuing a discovery program for ALK7 antagonists.
Disrupting AURKA's active conformation to treat MYCN-driven cancers; inhibiting ERdj3 to treat Gaucher's disease; using noninvasive electromagnetic stimulation to improve associative memory; and more...
Activity-directed synthesis of bioactive scaffolds; CRISPR-Cas9 genome editing for latent viral infections; nanoparticles encoding the AEG1 promoter for prostate cancer imaging; and more...