Caribou Biosciences’ CEO discusses CRISPR progress, future goals, and gender equality in biotech

Founded in 2011, Caribou Biosciences is a pioneer in the development of CRISPR genome editing technologies, a field honored with the Nobel Prize in Chemistry in 2020. Co-founded by Jennifer Doudna, Ph.D., one of the Nobel laureates, and CEO Rachel Haurwitz, and two other CRISPR pioneers, the company has raised over $800 million in funding, including significant investments from industry giants like Pfizer. In 2021, the company entered the clinic with their lead program and completed a successful $350 million IPO.

“Today, we’re laser focused on using Caribou’s next generation CRISPR technology, which we call chRDNA technology to advance a pipeline of wholly owned off the shelf CAR-T and CAR-NK cell therapies,” said Haurwitz in a recent interview at the JP Morgan Health Care conference.

CRISPR: From lab to clinic in about a decade

Rachel Haurwitz, Ph.D., CEO of Caribou Biosciences

Rachel Haurwitz,…

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Using AI to unlock new uses for existing cancer medicines

[LariBat/Adobe Stock]

Repurposing is a drug development strategy that has been widely applied in cancer. This strategy, sometimes called label expansion, involves obtaining FDA approval to market a drug for the treatment of new indications, alone or in combination with other drugs. Not only can this approach extend the window of patent protection for a commercialized drug, but the path to regulatory approval can also be far less costly and risky than the traditional approach to drug development, largely because clinical evidence of safety already exists. And most importantly, the result is more and better treatment options for patients.

In keeping the law of unintended consequences, recent provisions introduced by the 2022 Inflation Reduction Act (IRA) blunted the potential benefits of drug repurposing. As a result of limiting the window of time during which pharmaceutical companies can recoup investments before…

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50 of the best-funded biotechs of 2023

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As the year draws to a close, it is clear that molecular science and diagnostics is the hottest funding area in the biotech industry. In an analysis of 50 of the best-funded biotechs of 2023 focused on human health, molecular and science and diagnostics startups collectively attracting roughly $945 million, dwarfing the figures in other segments. The next popular two niches, gene therapies and oncology, had average funding levels of approximately $245 million and $170 million, respectively. While AI has received a significant amount of attention this year, biotechs specializing in that field garnered an average funding of only about $66 million. Outside of the life sciences, startups with a broader focus on AI raised a cumulative average of $202.47 million, based on an analysis of close to 1000 companies.

Caris Life Sciences has raised nearly $1.7B to date

In terms of best-funded companies overall,…

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Insilico Medicine taps AI to nominate small molecule inhibitor ISM9274 as a preclinical cancer therapy

Clinical-stage AI company Insilico Medicine has nominated a novel small molecule inhibitor known as ISM9274 as a preclinical candidate for cancer treatment.

The company used its PandaOmics AI platform to analyze genomic data from more than 90 tumor types and identified CDK12 as a promising target for multiple cancers including triple-negative breast cancer, lung cancer, and pancreatic cancer.

Next, it used its AI drug discovery engine Chemistry42 to design ISM9274 to selectively inhibit CDK12 and CDK13. Preclinical studies showed ISM9274 demonstrated potent antiproliferative activity across 60 cancer cell lines representing 13 tumor types. It also showed efficacy in animal models as both monotherapy and in combination with other therapies.

“Our target discovery philosophy is to find an optimal balance between commercial tractability, novelty and confidence,” said Alex Zhavoronkov, founder and CEO of Insilico Medicine.

The company’s AI platfo…

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AbbVie and Janssen receive positive CHMP opinions for novel cancer therapies

Epcoritamab Image courtesy of Genmab

The European Medicines Agency (EMA) could soon change the face of cancer treatment with its potential approval of two novel bispecific antibodies, epcoritamab and talquetamab. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting conditional marketing authorization for two novel cancer therapies — AbbVie’s epcoritamab and Janssen’s talquetamab.

Epcoritamab for relapsed/refractory diffuse large B-cell lymphoma

Epcoritamab is an investigational bispecific antibody for adults with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. CHMP made the decision after reviewing data from the pivotal EPCORE NHL-1 phase 1/2 trial showing an overall response rate of 63.1% with epcoritamab monotherapy in DLBCL patients. If approved, epcoritamab would be the first subcutaneous bispecif…

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AdAPT-001 oncolytic adenovirus shows promising phase 1 cancer treatment results

Rendering of AdAPT-001

Oncolytic adenoviruses have won significant attention in recent years as a novel approach to cancer treatment. One example of the trend is AdAPT-001 TGF-ß Trap, an engineered variant of the common cold virus equipped with a transforming growth factor-beta (TGF-β) “trap.” This mechanism is designed to latch onto and neutralize TGF-β, an immunosuppressive cytokine involved in cancer cell growth and disease progression.

At the 2023 ASCO Annual Meeting, the developer of AdAPT-001, EpicentRx, shared initial findings from a phase 1 trial on AdAPT-001. The initial data indicates that patients generally tolerate AdAPT-001 well.

So far, the study has enrolled 28 patients for treatment with AdAPT-001 as a single agent. EpicentRx plans to enroll an additional roughly 50 patients for a phase 2 portion where AdAPT-001 will be combined with immunotherapy.

In the phase 1 por…

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