FDA Law Blog

JANUARY 4, 2023

The purpose of these town halls are to discuss topics related to OTAT-regulated products, engage with product development stakeholders, and to provide information to help stakeholders to help advance drug development. The next town hall will focus on the clinical development of gene therapy products for rare diseases in February 2023.

Reagent 59

Reagent Manufacturing Production Gene Therapy 59

Camargo

MAY 2, 2021

A 2020 PhRMA report on the cell and gene therapy pipeline found 362 therapies in a range of stages in clinical development by biopharmaceutical companies in the United States. Source: Medicines in Development | 2020 Update: Cell and Gene Therapy , PhRMA. Identify critical development issues or deficiencies to address.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

The Pharma Data

DECEMBER 2, 2020

ERS Genomics – Ireland’s ERS Genomics Limited, and Germany’s Vivlion GmbH, announced a non-exclusive license agreement granting Vivlion access to ERS Genomics’ CRISPR/Cas9 patent portfolio, to enhance Vivlion’s gene editing reagents and screening services. to establish a new joint venture company, Nanjing AuroRNA Biotech Co.,

Drugs 52

Drugs Gene Editing Genome Genomics 52

Advarra

NOVEMBER 29, 2022

Bottlenecks in the manufacturing process, supply chain issues such as accessibility of good manufacturing practice (GMP)-grade reagents, and shortages of qualified scientists and engineers have caused many therapies to fail at critical stages of the clinical development pipeline.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

The Pharma Data

OCTOBER 14, 2020

Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML. ERS Genomics – Ireland’s ERS Genomics signed an agreement with FASMAC Co.,

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing In-Vivo 52

XTalks

JANUARY 6, 2025

As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas. While these therapies span all stages of clinical development, they are particularly concentrated in earlier phases, indicating strong future growth. billion by 2031.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

XTalks

MARCH 11, 2025

The Current State of mRNA Analytics There isnt a lot of emphasis right now being put on new method development specifically for application to multivalent mRNA vaccines. And I think this is surprising given just how many multivalent mRNA vaccines are currently in clinical development, begins Dr. Dawson.

Vaccine 64

Vaccine Vaccination Development Manufacturing 64