Keyword: BioMarin Pharmaceutical
BioMarin’s CSO Lon Cardon, Ph.D., is now the rare disease specialist’s chief scientific strategy office, expanding his purview beyond research.
BioMarin dropped a lower dose of its treatment, amid plans to get its applications into the hands of regulators before the end of the year.
The update adds to the impression that the gene therapy is a threat to assets including BioMarin’s valrox and Spark Therapeutics’ SPK-8011.
An Italian team has developed a viral vector for hemophilia gene therapy and shown in monkeys that it may improve efficacy.
Palynziq is a next-generation rival for BioMarin’s older drug Kuvan, which is licensed for PKU but requires patients to adhere to a restrictive diet.
Spark Therapeutics’ hemophilia A gene therapy reduced bleeding by 97% in a phase 1/2 trial but sent one patient to the hospital.
BioMarin teamed up with a senior center near its California HQ to turn an environmental cleanup site into new R&D space and housing for the elderly.
The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.
As the dust settles on the eye-watering $14 billion buyout from Pfizer for the one-marketed-drug biotech Medivation, other mid-cap companies are now sitting pretty and increasingly hopeful they will see suitors lining up to also bet big on biotech.
A rumor that BioMarin will be bought by Roche has popped up again, sending the biotech’s shares climbing fast with it.