News
The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia.
FEATURED STORIES
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator.
Job Trends
Merck, known as MSD outside of the United States and Canada, announced that the company will initiate a new Phase 3 clinical program with once-daily islatravir for the treatment of people with HIV-1 infection.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Unpredictable communication and a lack of transparency are eroding the industry’s and the public’s trust. The FDA, experts agree, needs to take control of the narrative.
THE LATEST
Already approved in schizophrenia and bipolar depression, Intra-Cellular Therapies reported strong late-stage data Tuesday for its antipsychotic Caplyta in major depressive disorder.
Despite the strong performance of its cancer portfolio, Johnson & Johnson on Tuesday reported Stelara sales of $2.45 billion in the first quarter of 2024, falling short of Wall Street expectations of $2.6 billion.
In a Phase I/II study, Ultragenyx’s antisense oligonucleotide GTX-102 led to significant cognitive, behavioral, sleep and gross motor improvements in patients with the rare genetic disorder Angelman syndrome.
A federal court in Massachusetts has granted Pfizer and BioNTech’s motion to put on hold Moderna’s lawsuit over alleged patent infringement related to their COVID-19 vaccine Comirnaty.
Prescriptions for Sandoz’s Hyrimoz, a biosimilar version of AbbVie’s Humira, have shot up after CVS Caremark took the blockbuster arthritis treatment off its major national commercial formularies on April 1.
While Madrigal Pharmaceuticals secured the first FDA drug approval for metabolic dysfunction–associated steatohepatitis, Akero Therapeutics is developing what may serve as a viable alternative treatment for precirrhotic disease.
Roche said Monday that its bispecific T cell engager Columvi improved survival in a Phase III lymphoma trial, clearing the drugmaker to seek a full, expanded label that could drive sales growth.
Following cases of convulsions in rabbits in a preclinical study, the FDA has placed a clinical hold on Neumora Therapeutics’ Phase I schizophrenia drug candidate NMRA-266.
Roche’s Genentech subsidiary is terminating for undisclosed reasons its 2021 contract with Adaptimmune for the development of allogeneic T-cell therapeutics.
AbbVie’s small molecule drug Qulipta has durable benefits and can reduce migraine frequency through 48 weeks of follow-up, according to interim Phase III data released on Friday.