Cures and Inspirations

June 7, 2023 / by Max Sellman

ASGCT highlights transformational approaches

When it comes to large events, the best place to be is front and center. And at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, that’s where we were, right inside the door in the middle of the exhibition hall. Not only were we able to greet nearly everyone who came to the exhibits, for the first time we joined other Danaher companies in one centralized location, allowing us to jointly highlight the Danaher mission, helping people realize life’s potential, to clients, partners and the industry in general.

Phenomenal initial results
I had the pleasure of attending several interesting talks during the conference. Some of the most exciting data were presented by Dr. Haydar Frangoul, who gave a clinical update on the efficacy of exa-cel from Vertex/CRISPR Therapeutics. Exa-cel, formerly known as CTX001™, is a cell therapy for mutations in the human beta globin (HBB) gene. Patients with HBB mutations experience a variety of disorders of the red blood cells including Sickle Cell Disease and β-Thalassemia. The standard of care for these conditions is blood transfusions and pain management; a cure has been elusive to date.

Vertex recently wrapped up a round of trials for exa-cel for 75 patients with Transfusion-Dependent β-Thalassemia (TBT) and Severe Sickle Cell Disease (SCD). Two separate patient pools received a single dose of exa-cel and saw a substantial increase in levels of fetal hemoglobin, which replaces the function of HBB.

More than 95% of TBT patients have been transfusion-free since treatment, and all of the treated SCD patients have avoided the vaso-occlusive crises that are typical symptoms of sickle cell. All indications so far are that these patients have been effectively cured of their genetic disorders with this cell therapy.

Collaboration key to cures
I also sat in on an inspiring presentation with Fyodor Urnov of the Innovative Genomics Institute. Fyodor highlighted some of the fantastic early-stage data that has come out of clinical trials, indicating that ~250 patients have been treated to date with CRISPR-based medicines. He emphasized that developers, manufacturers, and regulators will have to work together to treat patients faster and at lower cost in order to fully realize the transformational benefits of CRISPR. It is good that we are moving deliberately with a focus on safety, but we need to remember that potentially curable patients are dying while we work through regulatory hurdles.

Fyodor reviewed some challenges with current regulatory frameworks and the tensions created by working within these frameworks. He ended by pitching an ambitious goal: we should seek to be able to go from patient identification to patient dosing within a 3-6 month window. To achieve this, the field will have to align around standard tools and resources that can be applied quickly to streamline regulatory review and approval.

Working within the industry and the regulatory agencies, Aldevron and other Danaher companies are excited to deliver on these tools and services as we continue to serve those working hard to develop lifesaving cell and gene therapies.

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