PPD

OCTOBER 12, 2023

These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type. There are over 2,000 CGTs currently being evaluated in clinical trials, with more than 200 in Phase III and 10-20 per year estimated to come to market by 2025.

Laboratories 52

Laboratories Protein Expression Clinical Trials Pharmacokinetics 52

The Pharma Data

MARCH 8, 2021

We hope this Phase 2 trial, with a simplified schedule, will provide evidence that the investigational vaccine can be used in populations that are at risk of contracting Lyme disease, potentially including children age five years and older,” said Kathrin Jansen, Senior Vice President and Head of Pfizer Vaccine Research and Development.

Vaccine 52

Vaccine Disease Protein Expression Trials 52

The Pharma Data

DECEMBER 8, 2020

Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. 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. Nuance Pharma . Pear Therapeutics.

RNA 52

RNA Therapies Disease Protein Expression 52

The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

Clinical Trials 52

Clinical Trials Trials Disease Therapies 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. Several bioanalytical strategies for the quantitation of ASOs are available at Altasciences.

Drug Development 52

Drug Development Treatment Drugs FDA Approval 52

The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations. Roche is also deeply committed to addressing barriers to clinical trial participation and advancing inclusive research. Many people with NMOSD remain misdiagnosed and untreated.

Clinical Trials 52

Clinical Trials Treatment Disease Therapies 52

The Pharma Data

OCTOBER 28, 2020

In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. About Ultragenyx.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40