Regulatory
As Vertex and CRISPR Therapeutics await the monumental potential approval of exa-cel, bluebird bio, Iovance and Rocket Pharmaceuticals wait patiently in the wings.
The regulator has released Mersana Therapeutics’ antibody-drug conjugate XMT-2056 from its clinical hold, allowing the biotech to proceed with Phase I studies of the candidate with a lower starting dose.
The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.
Pfizer, Moderna, Novavax and more are working to combine preventative shots for COVID-19, flu, RSV and other illnesses. Will convenience be enough to overcome vaccine fatigue?
In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.
With the potential FDA approval of an MDMA-based therapy for PTSD on the horizon, biopharma stakeholders are eyeing psychedelics with fresh anticipation.
Protocol design optimization and timely engagement of regulators are the crux of optimized, patient-centric clinical trials.
Even after the runaway success of mRNA vaccines against COVID-19, the pathway to approval for upcoming would-be mRNA therapeutics has yet to be laid out.
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.
A Discussion with IQVIA’s Michelle Gyzen Sr. Director, Regulatory Affairs and Drug Development Solutions
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