Phase I
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.
High multiplexed patient-centric assays could reduce patient burden
Protocol design optimization and timely engagement of regulators are the crux of optimized, patient-centric clinical trials.
The regulator lifted a clinical hold on Verve Therapeutics’ CRISPR-based therapy clearing its Investigational New Drug application and plans to evaluate VERVE-101 in a Phase I heterozygous familial hypercholesterolemia trial.
Uniting thought leaders across the life sciences landscape, JLABS @ NYC rejoices in five years of catalyzing startups.
The biotech will pause its lirafugratinib program for a rare bile duct cancer to target a larger FGFR2-altered solid tumors population, citing the Inflation Reduction Act as a driving decision factor.
In a Phase Ib/II study, Tempest Therapeutics’ investigational PPAR⍺ antagonist—combined with Roche’s Avastin and Tecentriq—showed strong signs of survival benefits in liver cancer patients.
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