Newsletter | September 1, 2020

09.01.20 -- Inside The Potential Effect Of HTA Outcomes On The Time To Reimbursement

From The Editor

Greetings Cell & Gene readers,


In this comparison of health technology assessments (HTAs) and time to reimbursement (T2R) for orphan drugs (ODs) and therapies from four HTA agencies, specific objectives include analyzation of discrepancies in the recommendations from the HTA outcomes of ODs across markets, comparison of T2R and identification of what factors contribute to a longer T2R, and examination of a subgroup of gene therapies to identify any differences relevant to these innovative products.


Recently, Expression Therapeutics announced the appointment of Bill Swaney as its VP of manufacturing. I caught up with Swaney for his input on why Expression Therapeutics is building its own clean room and the benefits of doing so.


Erin Harris, editor in chief
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Industry Insights
Built To Fail: How Today’s Manufacturing Options Leave Pharma At Risk

Though pharma companies do their best to understand the environment, it can be difficult to predict demand, and the consequences of incorrect forecasts can be very costly in a number of ways. 

Reducing Cost Of Goods For Gene Therapy

Frost & Sullivan recently invited industry leaders with gene therapy experience to participate in a new thought leadership forum, our Virtual Think Tank. This forum brought together leading minds in this emerging field to discuss key challenges and other insights related to reducing the cost of goods (COGs) for gene therapy.

Distributed Biobanking: A Solution For FFPE Tissue Block Management

A new end-to-end system has been developed and implemented in several locations worldwide to address the issues encountered with formalin-fixed paraffin-embedded (FFPE) block storage, retrieval, and restocking.

In-House Versus Outsource: A Decision-Making Guide

Biopharma execs must consider how outsourcing fits into development and production strategies, and how to select the best CDMO partner for their business. Here’s a step-by-step guide to those decisions.

Improving Lentiviral Vector Downstream Processing Workflows

Lentiviral vectors (LVV) are a common vehicle to deliver genetic material in CAR T-cell therapy and gene therapy applications. Production methods have been developed by adapting technologies from the bioprocessing sector. However, these downstream workflows are long, require substantial manual labor, and suffer from low yields of infectious virus. In this article we discuss process development pain points of a modern workflow and ways to address them.

Thinking Outside Of The Freezer: DNA And RNA Storage

While ultra-low temperatures are the current state-of-the-art solution for biospecimen storage, ambient storage is an interesting option and could be superior in certain limited situations. For many types of samples, it’s possible that we will always have to use ultra-low temperatures for long-term storage. For other sample types, such as DNA and RNA, there are alternative solutions.  

Assessing The Impact Of COVID-19 On The Cell And Gene Industry

In May 2020, G-Con conducted a survey to assess the impact of COVID-19 on the biopharmaceutical industry. We're giving you a summary of the results in the infographic. How does this compare to your organization's experience?

Process Development Is Key To Patient Success

Process development (PD) is a critical component to the commercialization of viral vector-based therapies. While some gene therapy developers may want to speed through this operation to meet deadlines, doing so can put product quality, safety, and efficiencies at risk.

Cell And Gene Therapy And COVID-19: New Opportunities For Global Collaboration

Dr Michael May, president and CEO, CCRM (Canada), discusses the impact of COVID-19 on the regenerative medicine sector, and how the current scenario fosters new collaborative models in a post-COVID-19 world for all stakeholders, at the inaugural Business of Regenerative Medicine Asia Pacific Symposium (Virtual) co-hosted by CCRM Australia and the NSW Stem Cell Network.