Newsletter | August 22, 2020

08.22.20 -- Inside FDA's Extension of Enforcement Discretion Period for Regenerative Medicine Products

From The Editor

Hello Cell & Gene readers,


Learn how Locanabio is building on the clinical success of antisense oligonucleotides (ASOs) and small interfering RNs (siRNA) by expanding and improving on the current approaches for modifying dysfunctional RNA to treat devastating genetic diseases.


Human cells, tissues, and cellular and tissue-based products (HCT/Ps) have emerged as an exciting and lucrative area of regenerative medicine, and the regenerative medicine industry has paid close attention to the FDA and its regulatory practices. This article discusses the pros and cons of the extension of the FDA’s enforcement discretion.


Erin Harris, editor in chief
Follow me on Twitter

Industry Insights
Solutions To Today’s Biomanufacturing Challenges

With more complex molecules in development, changing capacity needs, uncertain forecasts, and increased competition, the market demands flexibility and innovative approaches to today’s challenges.

The Benefits Of Flow Cytometry In Immuno-Oncology Research

Flow cytometry introduces a powerful and versatile technology into studies, enabling in-depth analysis of cell signaling and behavior.

Understanding Recent Regulatory Changes For Pediatric Oncology Trials

While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for innovative therapies for pediatric cancers is becoming increasingly vocal.

Managing Refrigerated vs. Frozen Drug Product: What’s The Big Deal?

The most common storage is ultra-low temperature storage, but the fastest-growing is cryogenic, in vapor phase liquid nitrogen. This transition is huge. The chain of temperature and custody data is imperative.  

Updated U.S. FDA Guidance For The Gene Therapy Industry: Key Changes And Areas Of Increased Focus

In January 2020, the United States Food and Drug Administration (FDA) released several new or updated guidance documents pertaining to human gene therapy products. This paper summarizes several key changes that will impact developers of gene therapies.

Tufts Study: Advantages Of Single-Source Drug Development, Manufacturing

A recent study by the Tufts Center for the Study of Drug Development (CSDD) compared cycle times and development economics between multi- and single-source CDMO models.

Got Data? Using Informatics To Protect And Improve Your Business

Life sciences companies are now seeing the benefits from informatics in the manufacturing side of the business. Are you ready to use data and analytics to improve decision-making? There are things you can do now to get started.

Process Economic Simulation For Scalable Production Of Adenovirus

This novel process was compared with a reference process in both stainless-steel and single-use configurations across various production scales and scenarios. Configurations using single-use equipment wherever possible were more cost-effective than configurations using primarily stainless-steel equipment.

The Path To Confidence At Every Point Of Connection

Practical information on key issues, including extractable/leachable testing in components, freeze/thaw in single-use assemblies, efficiencies driven by SUT advances such as genderless connectors, and more.

Unlocking The Evidence-Generation Potential Of The EMR

We have only just begun to scratch the surface of the potential of the electronic medical record (EMR) to support research and evidence generation in the realm of drug development. During this discussion, we examine several practical applications of real-world data from EMRs to support research.

Planning For Commercial Success: Best Practices For Designing Cell And Gene Therapy Clinical Trials

Cell and gene therapy (CGT) applications have, in the last three years, made the jump from pure research products to commercialized products with high clinical efficacy, particularly for hematologic malignancies. This webinar elaborates on how to design early development programs and clinical trials for CGTs to ensure that the product can be commercialized successfully in all target markets, i.e., can be reimbursed and adopted successfully despite small patient numbers, rare diseases, complex administration, etc.