Advancing Gene-Editing in Cancer Treatment: Caribou’s CAR-T Therapy Shows Promise

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Investors initially had high hopes for Caribou Biosciences, a gene-editing company that went public in 2021. However, as enthusiasm for genetic medicine start-ups dwindled, Caribou stock experienced a significant decline, dropping from over $30 to a recent low of $4.

In a surprising turn of events, Caribou (CRBU) emerged as one of the market’s top gainers on Thursday, surging by an impressive 46% to reach $5.94, thanks to Pfizer’s (PFE) acquisition of a 7% stake in the company. As part of the deal, Pfizer secured an option for one of Caribou’s cancer programs. Although Pfizer’s investment of $25 million may appear modest, it signifies a renewed interest in gene-editing technology.

Caribou is among the public biotech firms currently conducting trials for medical treatments based on Crispr, a pioneering technique that earned its discoverers the Nobel Prize. Crispr allows scientists to target specific problematic sections of cellular DNA and make necessary alterations. Similar to Caribou, stocks of other Crispr-based companies like Crispr Therapeutics (CRSP) and Intellia Therapeutics (NTLA) have seen a resurgence this year, demonstrating promising results in the treatment of cancer and genetic disorders through their clinical trials.

According to Rachel Haurwitz, the CEO of Caribou, investors are now eagerly awaiting clinical trial data that will determine whether Crispr editing truly provides tangible benefits to patients.

Currently, Caribou is conducting early trials for two treatments. Their investigational therapy, CB-010, aims to combat lymphoma, a type of blood cancer, by using genetically modified immune cells that specifically target the cancerous cells. While three CAR-T therapies from other pharmaceutical companies have already been approved for use, these therapies involve modifying a patient’s own immune cells. Unfortunately, not all patients’ cells are suitable for CAR-T modification.

Caribou Biosciences is a frontrunner in the field of gene editing, and with ongoing trials and investments from key players like Pfizer, the company is set to shape the future of medicine and genetic therapies.

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Advancing Gene-Editing in Cancer Treatment: Caribou’s CAR-T Therapy Shows Promise

Caribou’s revolutionary CAR-T therapy employs gene-editing techniques to modify immune cells derived from healthy donors. The use of off-the-shelf CAR-T cells holds the potential for enhanced effectiveness, as evidenced by the initial results seen in the first six lymphoma patients treated with this ground-breaking therapy.

One of the most remarkable outcomes observed thus far is the complete disappearance of cancer indications in all six patients following their initial treatment with Caribou’s CAR-T therapy. While it is important to note that this does not guarantee a permanent remission, two patients have displayed sustained positive responses for a minimum of one year. In the latter half of this year, Caribou plans to release the six-month results for all patients, providing further insight into the therapy’s long-term efficacy.

There are several companies actively engaged in developing off-the-shelf CAR-T cell therapies. However, Caribou’s early outcomes stand out as some of the most promising in the field.

As a part of its second clinical trial, Caribou is currently evaluating an off-the-shelf CAR-T treatment for multiple myeloma, a type of blood cancer. It is worth noting that Legend Biotech (LGEN) and Johnson & Johnson (JNJ) gained approval for a CAR-T therapy involving genetically modified cells from individual patients just last year.

Pfizer has expressed particular interest in Caribou’s off-the-shelf CAR-T therapy for multiple myeloma, referred to as CB-011. Through its investment, Pfizer secures the right to negotiate a license for CB-011 if another external organization expresses an intent to acquire the product developed by Caribou.

According to CEO Haurwitz, current self-donated CAR-T cell therapies are inaccessible for many cancer patients. The development of off-the-shelf products, such as the ones being pioneered by Caribou, holds the promise of making CAR-T treatments more widely available and, hopefully, more efficacious.

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