From The Editor | December 10, 2019

First-In-U.S. Trial Of CRISPR-Edited Immune Cells


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


According to last month’s announcement, the first-in-U.S. trial of CRISPR-edited immune cells for cancer patients suggest safety of approach. Genetically editing a cancer patient’s immune cells using CRISPR/Cas9 technology, then infusing those cells back into the patient appears safe and feasible based on early data from the first-ever clinical trial to test the approach in humans in the United States. Researchers from the Abramson Cancer Center of the University of Pennsylvania have infused three participants in the trial thus far — two with multiple myeloma and one with sarcoma — and have observed the edited T cells expand and bind to their tumor target with no serious side effects related to the investigational approach. Penn is conducting the ongoing study in cooperation with the Parker Institute for Cancer Immunotherapy (PICI) and Tmunity Therapeutics, a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy to patients with devastating diseases. I caught up with Usman “Oz” Asam, President and CEO at Tmunity for more information, and here’s what he had to say.

  • What is Tmunity Therapeutics’ role in the collaboration with the University of Pennsylvania for the first-in-U.S. trial of CRISPR-edited immune cells for cancer patients?

The trial was conducted at the University of Pennsylvania. UPenn, Tmunity Therapeutics and the Parker Institute of Cancer Immunotherapy (PICI) provided financial support for the trial.

  • From Tmunity’s perspective, that does this Phase 1 study mean for the sector in terms of safety?

We cannot make a broad statement on the safety of this therapy until the trial completes. While this phase 1 safety study continues and genetic analysis is ongoing, the team conducting the trial sees the feasibility of the trial and safety so far provides optimism that the approach may be applicable across multiple areas of gene therapy research.

  • Why this study is ground-breaking for the sector?

These data are from the first-in-US trial of a CRISPR-edited immune cell therapy. For Tmunity, it established a beachhead in the continued evolution of our innovative portfolio and provides key insights into the development of allogeneic cell therapies.

  • What is on the horizon for Tmunity?

We continue to screen and enroll patient in our phase 1 trial with our PSMA CAR-T therapy in metastatic castrate-resistant prostate cancer. We are also opening our second, multi-center PSMA-02 trial in the same patient population.

We are screening patients in a phase 1 trial with TnMUC1 CAR-T — a previously undruggable target — in a multi-center trial that will be open to patients with non-small cell lung cancer, ovarian cancer, triple negative breast cancer, pancreatic cancer and multiple myeloma. We hope to have preliminary data on both these trial by YE2020.