Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

Vaccine 52

Vaccine Therapies Clinical Trials Small Molecule 52

The Pharma Data

APRIL 27, 2022

The Food and Drug Administration’s (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. For more information about the trial, visit ClinicalTrials.gov. Enhertu Enhertu is a HER2-directed ADC.

Therapies 52

Therapies Trials Protein Expression Treatment 52

The Pharma Data

SEPTEMBER 28, 2021

Also, the info from SRP-9001 have helped to optimise the planning of the upcoming phase III clinical trial trial for DMD,” said Levi Garraway, M.D., quite 4,000 patients are treated with Evrysdi in clinical trials, compassionate use, and real-world settings. Roche’s Chief medic and Head of worldwide development.

Clinical Trials 52

Clinical Trials Protein Expression Therapies FDA 52

Alta Sciences

APRIL 17, 2024

Food and Drug Administration (FDA) as a nasal spray called esketamine, for treatment-resistant depression. They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify protein expression.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 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

JANUARY 13, 2021

we determined the crystal structure of CD40-ligand 2 , developed a humanized version of our antibody (hu5c8, ruplizumab, or Antova®) and tested it in human trials for preventing organ transplant rejection and autoimmunity. CD40-ligand is a protein expressed on the surface of activated T lymphocytes that mediates T cell helper function.

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

The Pharma Data

JANUARY 15, 2021

In the DESTINY-Gastric01 trial, patients (n=126) in the Enhertu treatment arm had a 41% reduction in the risk of death versus patients (n=62) treated with chemotherapy (based on a hazard ratio [HR] of 0.59; 95% confidence interval [CI] 0.39-0.88; Regular approval by the U.S. months [95% CI 9.6-14.3] months [95% CI 6.9-10.7] with chemotherapy.

Treatment 52

Treatment Trials Disease Therapies 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. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021. Pear’s reSET, reSET-O and Somryst are the first PDTs to receive FDA approval for treating disease.

RNA 52

RNA Therapies Disease Protein Expression 52

The Pharma Data

SEPTEMBER 10, 2020

OCREVUS is the first and only treatment approved for both relapsing MS (RMS) and primary progressive MS (PPMS) and now more than 170,000 people have been treated with OCREVUS globally in clinical trial and real-world settings; favourable benefit-risk profile remains consistent over 7 years. and primary progressive MS (PPMS).

Treatment 52

Treatment Disease FDA Approval Clinical Trials 52

The Pharma Data

OCTOBER 28, 2020

Food and Drug Administration (FDA) prior to resuming enrollment and dosing. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Food and Drug Administration (FDA). About GeneTx Biotherapeutics.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

Conversations in Drug Development Trends

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Initial approval in 1994: FDA, U.S.

Treatment 81

Treatment Clinical Trials Small Molecule Therapies 81

The Pharma Data

DECEMBER 3, 2020

Clinical Trials. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Na V 1.1 December 6, 2020 12:00 PM – 1:30 PM; Track: 7.

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

The Pharma Data

JANUARY 13, 2021

However, before the Phase III program may begin, the FDA requested a meeting with Theratechnologies to discuss questions and comments received on certain aspects of the proposed trial design, to ensure alignment with the agency’s expectations with NASH trials. Vivity received FDA approval in February 2020.

Licensing 52

Licensing Licensing Clinical Trials Trials 52

The Pharma Data

OCTOBER 26, 2020

The purpose of this exploratory Phase 1b/2 trial was to evaluate the safety and efficacy of the combination of TG4001 and an immune checkpoint inhibitor in a heterogeneous group of patients with aggressive, recurrent and/or metastatic HPV16-positive cancers. Key findings of the trial: .

Vaccine 40

Vaccine Trials Treatment Virus 40

The Pharma Data

SEPTEMBER 22, 2020

Basel, September 23 , 2020 – Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (SMA).

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 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.

Clinical Trials 52

Clinical Trials Disease Treatment Therapies 52

The Pharma Data

APRIL 8, 2021

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

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. Section #1: FDA Interactions Given the wide range of sponsors (i.e., Where vector presence is detected, transgene mRNA and/or protein expression levels should also be measured.

Therapies 59

Therapies FDA Production Disease 59

Drug Target Review

JANUARY 20, 2025

The oncology drug development landscape is evolving rapidly, driven by the deployment of targeted therapies in precision medicine and regulatory initiatives like the FDAs Project Optimus. Molecular factors Molecular markers, such as protein expression and signalling pathway activation, also guide therapy choices.

Clinical Trials 52

Clinical Trials Trials Drugs Drug Development 52