The Pharma Data

SEPTEMBER 28, 2021

In addition, results from Part 1 of Study SRP-9001-102, an ongoing, randomised, double-blind, placebo-controlled clinical test evaluating the security , efficacy, and tolerability of one dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary biological endpoint of change in micro-dystrophin protein expression from baseline.

Clinical Trials 52

Clinical Trials Protein Expression Therapies FDA 52

The Pharma Data

DECEMBER 8, 2020

CEO and founder Min Li knows a thing or two from his time running GSK’s neuro R&D group before the company shuttered the program in 2017. As we reach the limits of what is easily druggable with protein targeting therapies, reprogramming RNA processing represents an exciting new therapeutic opportunity. Pear Therapeutics.

RNA 52

RNA Therapies Disease Protein Expression 52

Alta Sciences

APRIL 17, 2024

The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A. Asia, and Europe.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

The Pharma Data

SEPTEMBER 2, 2020

New Phase III data from SAkuraStar and SAkuraSky studies demonstrate reduced severity of relapses with ENSPRYNG (satralizumab), recently FDA-approved as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).

Clinical Trials 52

Clinical Trials Disease Treatment Therapies 52

The Pharma Data

APRIL 8, 2021

Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. EVRYSDI was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

OCTOBER 26, 2020

Increased infiltrates, expression of immune related genes and higher PD-L1 protein expression were observed across all patients suggesting a remodeling of the tumor microenvironment consistent with a switch toward a “hot tumor” phenotype. Avelumab Important Safety Information from the US FDA-Approved Label.

Vaccine 52

Vaccine Trials Treatment Virus 52