
Feature|Videos|December 2, 2024
Rare disease populations as pivotal proving grounds
Repairing nucleotide abnormalities for rare disease populations as pivotal proving grounds for transformative therapies.
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Episodes in this series
In this exclusive BioPharm International Peer Exchange video series, Chris Spivey, Director of Industry Relations and Strategic Partnerships, talks with industry experts about various aspects of Cell and Gene Therapies with a focus on reducing manufacturing bottlenecks by creating smaller automated footprint installations, better integrating analytics advances including harnessing artificial intelligence, and above all lowering barriers to access by lowering costs while improving timelines.
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FDA expands Winrevair’s label with HYPERION data, formalizes the use of non-animal testing methods, and Amgen reports positive Phase 3 results for dazodalibep in Sjögren disease.

The FDA has approved a label update for Merck's Winrevair (sotatercept-csrk) incorporating Phase 3 HYPERION data, showing a 76% reduction in clinical worsening events when added to background therapy in adults diagnosed with pulmonary arterial hypertension within the past year.

BIIE CEO Dr Stephen Wilson outlines rapid growth plans and a multidisciplinary technology stack targeting global clinical translation.

FDA's new rule formally recognizes non-animal methods like organoids and AI models as valid ways to support human drug testing.

Dazodalibep hit its phase 3 primary endpoint at week 48, building on phase 2 data showing a 6.3-point ESSDAI reduction versus placebo.

Glycomine's GLM101, an investigational liposomal mannose-1-phosphate substrate replacement therapy, has received FDA Breakthrough Therapy designation for PMM2-CDG, a rare glycosylation disorder with no approved treatments.

How early Phase I decisions can shape or compromise the regulatory and financing outcomes that define a biotech program's future.
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