Home Insights What’s new in the field of hematologic cell and gene therapy: Part I

What’s new in the field of hematologic cell and gene therapy: Part I

Julianne Dunphy, PhD, Global Director, Medical Strategy at Mind+Matter, part of EmerGENE, shares a series of standout findings presented at the 63rd American Society of Hematology (ASH) annual meeting and exposition in December 2021.

The largest conference of its kind in the world, ASH is attended by more than 25,000 industry professionals from across the globe. Taking place over four days, the conference saw the presentation of independent academic studies as well as industry-sponsored research and developments exploring a wide array of malignant and nonmalignant hematologic diseases, from advances in basic science to readouts of large clinical trials.

As in previous years, some of the most notable discussions featured developments in the field of cell & gene therapy (C&GT) that have far-reaching implications for the treatment of hematological conditions. While there were many notable topics discussed at ASH, in this blog Julianne explores a topic with the potential to expand the possibilities of C&GT as we know it.

Off-the-shelf CAR-T therapies: the future could look a bit different than originally imagined

Autologous CAR-T cell treatments are, by definition, created for each individual patient, which demands not only careful patient selection but, importantly, a lengthy and costly manufacturing process prior to administration. For these and other important reasons, allogeneic or “off-the-shelf” options are a hot area of research and product development.

An oral session at ASH, with a focus on allogeneic CAR therapies, provided an interesting sample of data that indicate promise but also raise questions about when and how they may fit into the treatment paradigm. Several companies and research groups are pursuing allogeneic CAR-Ts, notably CRISPR Therapeutics, Allogene, and Precision Biosciences, among others. Data were reviewed with the latter’s treatment in development, PBCAR0191, in a group of relapsed or refractory NHL and B-ALL patients[1]. While response rates were solid, the durability of effect was disappointing, with most patients relapsing by the 6-month time point.

They aren’t unique in their findings; other allogeneic treatments under study have also heralded durability concerns. One and done? Signs aren’t good on that front. But interestingly, the session also included consolidation dosing data on Allogene’s TALEN gene-edited CAR treatment ALLO-501A in patients with relapsed/refractory large B-cell lymphoma (LBCL)[2]. One group of patients received a single dose while patients with stable disease in the other group received a second dose, with the latter group experiencing improved response rates. These early data bode well for the possibility of redosing an “off-the-shelf” treatment.

What will this mean for patients?

These data and others highlight the questions that still need to be answered as well as suggest potential strategies for their use in practice. Might they be useful in those cases where autologous CAR-T therapies aren’t possible because of patient fitness or manufacturing failure? Might they be appropriate after autologous CAR-T in the relapse setting? Or are they more ideal for patients with extremely aggressive diseases that can’t wait?

The hope is that this next generation of CAR-T will mitigate key obstacles currently preventing patients from receiving treatment, primarily the issues of cost and time, while potentially broadening the eligible patient population. Having multiple options in different scenarios of treatment and relapse is always a win. As seen at ASH, research pioneering the advancement of these treatments still has a lot to sort out before we see these treatments in clinical practice.

Keep the conversation going

At EmerGENE, we are committed to exploring the latest advancements in C&GT to guide our customers in getting these life-changing treatments to patients. None of these studies could be done without the generous support and participation of the patients and caregivers and are the driving force behind what we do as well. For further insights, please click here.


[1] Nitin Jain, et al: 650 Preliminary Safety and Efficacy of PBCAR0191,an Allogeneic ‘Off-the-Shelf’ CD19-Directed CAR-T for Patients with Relapsed/Refractory (R/R) CD19+ B-ALL. Abstract available here: https://ash.confex.com/ash/2021/webprogram/Paper153166.html

[2] Lazaros J. Lekakis, et al: 649 ALPHA2 Study: ALLO-501A Allogeneic CAR T in LBCL, Updated Results Continue to Show Encouraging Safety and Efficacy with Consolidation Dosing. Abstract available here: https://ash.confex.com/ash/2021/webprogram/Paper146045.html


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