What a $2 million per dose gene therapy reveals about drug pricing
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...
Managed Healthcare Executive2d
FDA, EMA See Differences in Cell and Gene Therapy Trial Data
Researchers have suggested that uniform evidence standards and reporting requirements could help speed patient access to cell ...
US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
STAT1d
Pharmalittle: We’re reading about RFK Jr. targeting CDC and FDA advisers, a Pfizer gene therapy, and more
HHS Secretary Robert F. Kennedy Jr. is preparing to remove committee members who advise the government on vaccine approvals ...
stocknews1d
3 Gene Therapy Stocks With Game-Changing Potential
Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, ...
FiercePharma1d
Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
MeiraGTx rises after publication of positive data from gene therapy trial for eye disorder
MeiraGTx (MGTX) stock rose 7% after publishing promising results from the first human trial using gene therapy to treat AIPL1 ...
Pfizer stops commercialization of hemophilia gene therapy Beqvez
Pfizer said on Thursday it will stop global development and commercialization of its hemophilia gene therapy, Beqvez, citing ...
FierceBiotech1d
MeiraGTx gene therapy allows 11 blind children to see as biotech eyes accelerated approval
Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene ...
Pfizer Discontinues Hemophilia Gene Therapy Beqvez As Interest Wanes
According to multiple media reports on Thursday, Pfizer Inc. (NYSE:PFE) has terminated the global development and ...
CNN5d
What a $2 million per dose gene therapy reveals about drug pricing
Because his drug was a gene therapy, public grant money and private philanthropy played an especially central role, with the National Institutes of Health alone putting over $450 million into ...