Posted by on February 10, 2021 6:35 pm
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AbbVie exploring how CRISPR gene editing can improve cell therapies – BioPharma Dive

Dive Brief:

  • AbbVie and Caribou Biosciences said Wednesday they will work together to improve a certain kind of cell therapy, with the help of CRISPR gene editing technology.
  • CAR-T cell therapies are a relatively new form of treatment that uses engineered T cells to fight cancer. The cells come from one of two sources: either the cancer patient, or a donor. Though donor-derived cells offer valuable advantages, one of the drawbacks is the patient’s immune system can see them as intruders and attack. This problem is what AbbVie and Caribou hope to address with their new collaboration, which aims to engineer CAR-T cells that can withstand an immune response.
  • Per deal terms, the companies will research and develop two new CAR-T cell therapies directed at targets specified by AbbVie. The therapies will be made using Caribou’s genome editing and cellular technologies, which AbbVie now holds exclusive rights to for the selected targets. AbbVie also has the option to expand the deal to include up to two more CAR-T cell therapies. Caribou, meanwhile, will receive $40 million upfront and could take home another $300 million if certain milestones are hit.

Dive Insight:

CAR-T cell therapies are powerful treatments that offer hope to patients who’ve exhausted most other options. A pivotal study of Novartis’ Kymriah, for example, found 83% of patients with a form of hard-to-treat leukemia went into complete remission within three months of infusion. Kymriah was the first CAR-T cell therapy approved in the U.S., and has since been joined by Gilead’s Yescarta and Tecartus as well as Bristol Myers Squibb’s Breyanzi.

Though they’re effective for the leukemias and lymphomas they treat, all four therapies use patients’ own T-cells, which presents significant challenges. The cells must be extracted, shipped, engineered, returned and then re-infused, all within a tight window of roughly two to three weeks. Any setbacks or mistakes along the way could be life-threatening, too, given that the patients receiving these therapies are often very sick.

Some drug developers are trying to address these challenges by focusing on donor-derived, or “off-the-shelf,” T cell therapies. The idea is that the use of donor cells allows treatments to be prepared ahead of time, meaning they can be deployed much faster than their patient-derived counterparts.

Having treatments ready could also lower the risk of running into manufacturing problems, which has been a lingering issue with patient-derived CAR-T cell therapies.

Smaller biotechs like Allogene Therapeutics, CRISPR Therapeutics and Precision Biosciences have spearheaded the push into off-the-shelf research, and in turn caught the attention of big pharmaceutical companies.

In 2018, Pfizer traded a portfolio of assets related to CAR-T cell therapy to Allogene, taking a 25% stake in the biotech in the process. More recently, Bayer, Johnson & Johnson and Merck & Co. have bet on off-the-shelf immunotherapies.

AbbVie, which built a blockbuster oncology business around its blood cancer drug Imbruvica, is joining the fray as well with its Caribou partnership.

The companies agreed that Caribou will be responsible for select pre-clinical research, development, and manufacturing activities, with AbbVie reimbursing its partner. AbbVie will be in charge of all clinical development, commercialization, and manufacturing efforts.

In addition to the upfront and milestone payments, Caribou may also receive additional payments for commercial milestones as well as global tiered royalties on products stemming from the deal.

Published at Wed, 10 Feb 2021 17:21:42 +0000