Phase I
As companies clamor for a piece of the antibody-drug conjugate pie, experts pose the question: is it possible to replicate the success of Enhertu?
BioSpace takes a deep dive into five investigational therapeutic cancer vaccines that have recently shown efficacy in difficult-to-treat indications.
The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial shows it is still viable as a potential treatment for a fatal, rare genetic disease.
Successful drugs from Novo Nordisk and Eli Lilly are just the beginning of what one analyst says could be “the largest therapeutic class of drugs that the biopharma industry has ever seen.”
At its highest dose level of 608 mg, lepodisiran reduced lipoprotein(a) levels by a median of 94% after 48 weeks.
When given at potentially therapeutic doses, Verve’s base editor led to strong reductions in LDL cholesterol and PCSK9 levels in patients with heterozygous familial hypercholesterolemia.
To protect the central nervous system, the blood-brain barrier bars entry to around 98% of molecules—but approaches like Roche’s trontinemab could spell new hope in Alzheimer’s and beyond.
As Vertex and CRISPR Therapeutics await the monumental potential approval of exa-cel, bluebird bio, Iovance and Rocket Pharmaceuticals wait patiently in the wings.
A failed rare disease clinical trial brings social media expertise to the pharma industry and patient recruitment: A discussion with Bryan Manning, founder of Clinical Enrollment and Two Blind Brothers
A recent report by Tufts Medical Center reveals impressive approval rates for durable cell and gene therapies, but experts urge caution over persistent safety and accessibility concerns.
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