The Pharma Data

SEPTEMBER 2, 2020

Roche’s Chief Medical Officer and Head of Global Product Development. From the success of our first-in-class B-cell MS therapy OCREVUS, we are poised to continue advancing the science in MS with our new investigational BTK inhibitor fenebrutinib, and in NMOSD with the recent FDA approval of ENSPRYNG.”.

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.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

Drug Target Review

MAY 22, 2024

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.

Therapies 59

Therapies RNA Protein Expression DNA 59

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

The Pharma Data

JULY 21, 2021

Positive top-line results have already been reported for two Phase 2 clinical trials of VLA15 in over 800 healthy adults. VLA15 is the only active Lyme disease vaccine candidate in clinical development today, and covers six serotypes that are prevalent in North America and Europe. About VLA15.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Pharma Data

AUGUST 9, 2021

Enhertu is being further assessed in a comprehensive clinical development programme evaluating efficacy and safety across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers. 1 More than two million patients with breast cancer were diagnosed in 2020, resulting in nearly 685,000 deaths globally.

Trials 52

Trials Treatment Clinical Trials Protein Expression 52

The Pharma Data

JANUARY 15, 2021

Enhertu previously received Priority Review and Breakthrough Therapy Designation (BTD) in the U.S. 3 HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors including breast, gastric, lung and colorectal cancer. About the Enhertu Clinical Development Program.

Treatment 52

Treatment Trials Disease Therapies 52

The Pharma Data

JANUARY 13, 2021

Dr. Lederman continued, “Despite the recognized promise of anti-CD40-ligand mAb therapy, first generation anti-CD40-ligand mAbs were limited because their crystallizable fragment (Fc) domain interacted with a cell surface receptor called Fc?RII, RII, which resulted in an increased risk of thrombosis. International Immunol. (11):1583

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

Conversations in Drug Development Trends

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. A condition of approval is the completion of a Phase 3 study designed to evaluate pheno-conversion in SOD1 mutation carriers (ATLAS study) to confirm clinical benefit.

Treatment 81

Treatment Clinical Trials Small Molecule Therapies 81

The Pharma Data

OCTOBER 26, 2020

Responses were observed in all primary tumor types and across all lines of prior therapy. Responses were observed in all primary tumor types and across all lines of prior therapy. In particular, in the patient with CR, lesions disappearance was accompanied by the development of a strong T-cell response against E6 and E7.

Vaccine 40

Vaccine Trials Treatment Virus 40

FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

Therapies 59

Therapies FDA Production Disease 59