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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Lumen Bioscience cracks the code on spirulina as a biologics factory for c. diff, metabolic disease and more

Clostridioides difficile, commonly known as C. diff, is a significant health threat in the U.S. Recent estimates suggest that C. diff, a common bacteria, can cause infection in roughly 500,000 patients annually in the U.S., with around 30,000 of these cases resulting in death. “Actually, it’s more like 5 million when you think about it in terms of people at risk,” estimated Brian Finrow, founder and CEO of Lumen Bioscience, a biotech specializing in developing novel biologics. C. diff infection (CDI) is especially prevalent and dangerous in the immunocompromised elderly patients who are hospitalized or have been in nursing homes for long periods. “It’s a huge cost driver,” Finrow. Annual U.S. costs tied to CDI have eclipsed $6 billion per year. The infection is especially prevalent and dangerous in the elderly. Complicating matters further, recurrent CDI infections are common, affecting anywhere from 20% to 35% of patients with an initial infection.

A s…
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Moving beyond buzzwords: When will a rising AI tide lift all Big Pharma boats?

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For all of the talk about AI in drug discovery and development, few Big Pharmas are putting up big bucks in AI spending. A CRB survey from late 2023 painted a conservative picture: about half of drug developers planned on allocating between $1 and $10 million for data and AI projects over the next two years. A mere 3% reported budgeting more than $50 million. No companies surveyed had budgeted more than $100 million.

By contrast, Microsoft has invested more than $10 billion into OpenAI alone. Amazon invested $4 billion into Anthropic for minority ownership and a board seat, while Google invested $2 billion plus potentially hundreds of millions more into Anthropic and Character AI for AI research and development. In early 2023, IDC projected that cumulative AI spending would reach $154 billion by the end of the year — 27% higher than a year earlier.

While a growing number of pharma and…

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Could an ‘extreme’ hibernating ground squirrel unlock new obesity treatments?

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In late 2023, Eli Lilly, whose stock is now up close to 80% over the past year, inked a deal with the Emeryville, California–based Fauna Bio potentially worth $494 million that focuses on the discovery of novel drug targets for treating obesity. In 2020, Fauna entered into an obesity-focused collaboration with Novo Nordisk, Lilly’s primary rival in the obesity treatment market. Coincidentally, Novo Nordisk’s stock is up more than 50% over the past year, thanks in large part to strong sales of the GLP-1 drugs Ozempic and Wegovy.

In the hunt for the next obesity blockbuster, both Lilly and Novo Nordisk are turning to Fauna Bio’s expertise in extreme mammal genomics. Among Fauna Bio’s focus areas is the 13-lined ground squirrel, an example of the company’s emphasis on “extreme mammals.” The squirrels are known for their remarkable metabolic transformations during hibernation that make them…

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Antibody-drug conjugates, genomics, and more: Key trends at the JP Morgan Healthcare Conference

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The 2024 JPMorgan Healthcare Conference showcased the resilience and innovation driving the biotech sector, even as the sector has recently grappled with financial headwinds such as declining IPOs, layoffs, and tightening cash reserves. Companies are tightening operations while still pushing forward in developing novel technologies like machine learning for drug discovery, cell and gene therapies, synthetic biology, and unlocking large-scale genomic edits. The CDMO market is also showing signs of renewed growth in early 2024, with opportunities in areas such as advanced therapeutics, oncology, and injectables.

Biotech resilience emerging as a theme, but ‘flawless execution’ a need

“JPM underscored the resilience of biotechs amid economic challenges, emphasizing strategic priorities like bringing assets into the clinic and an unwavering commitment to timelines,” said M…

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Quantum promises a bright future for the pharma industry

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Quantum computing technology continues to advance closer to an everyday reality. While the potential applications of quantum in certain sectors are more speculative, the pharmaceutical industry is poised to be transformed by quantum technology.This sector is likely to be the fastest to unlock the benefits of quantum computing, once the technology is fully realized. But how ready are companies in this sector for this revolution, and what changes can we expect?

The next generation of molecular modelling

The key to discovering new drugs involves testing and understanding the reactions between different materials and catalysts. Rather than relying solely on experiments in labs, scientists today also use computer aided drug design and molecular modellng to simulate these interactions and accelerate the discovery of new compounds.

However, even today’s most powerful supercomputers…

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Deepcell’s CEO forges her own path to transforming cell science with AI and microfluidics

Deepcell co-founder Maddison Masaeli took an unexpected path from academic research to launching an AI-powered cell analysis platform. The company has raised close to $100 million in funding to support its mission. This includes a $73 million Series B round and an earlier $20 million Series A round. The company has entered into a research collaboration with NVIDIA and made generative AI a core focus of their research and development efforts.

From electrical engineering to AI-powered biology

The Deepcell CEO since the company’s founding, Masaeli’s journey shifted from her bachelor’s study of electrical engineering to the life sciences when a chance encounter brought her to a biotech lab as a Harvard-MIT research scholar. “I randomly got introduced to this amazing biotech lab at Harvard at a social event,” she recalled. “I decided to go and explore and see what that was all about.”

The lab specialized in developing methods for tissue engineering…

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Biotech bounces back at JPM 2024 on optimism, breakthroughs and calculated bets, but uncertainties persist

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At the dawn of 2024, there’s a sense of renewed optimism in the biotech sector despite a downturn that has lasted for more than two years. At the JP Morgan Health Care Conference, deal-making activity showed signs of strength. For instance, Merck agreed to acquire cancer drug developer Harpoon Therapeutics for roughly $680 million, highlighting continued interest in oncology cancer therapies. Meanwhile, Novartis announced plans to acquire Calypso Biotech, a deal involving an upfront payment of $250 million with potential milestones worth up to $175 million.

“I feel like in the last two or three weeks, we’ve almost made up for 50% of the deals that didn’t happen in 2023,” quipped Jen Nwankwo, CEO of 1910 Genetics, a company specializing in computational biology and automated laboratory technologies. Pointing to recent clinical successes and FDA approva…

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Recursion partners with NVIDIA to unveil Phenom-Beta, democratizing access to its $1 Billion phenomics investment

In sync with the JP Morgan Health Care Conference, Recursion Pharmaceuticals has unveiled Phenom-Beta, a deep learning model designed to transform cell microscopy images into meaningful biological representations. “Phenom-Beta allows you to take images of human cells from a microscope or other sources and turn them into mathematical representations of biology,” said Chris Gibson, CEO of Recursion, in an NVIDIA-hosted call with journalists.

Hosted on the NVIDIA BioNeMo platform, Phenom-Beta is the company’s first in a potential series of foundation models for external use. The name “Phenom-Beta” references both “phenomenal” and “phenomics.” The scientist Steven A. Garan coined “phenomics” in 1996 to refer to the study of phenotypes, the observable characteristics or traits of an organism influenced by genetic predisposition and environmental factors.

Recursion bets the new frontier in phenomics could echo the rise of genomics

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Amgen, Deepcell tap NVIDIA’s AI to drive drug discovery and advance cell morphology research

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In 2023, generative AI (GenAI) entered the mainstream, capturing the attention of both the public and a growing number of biotechs. One of the most prominent Big Tech firms driving the trend is NVIDIA, which has forged alliances with biotechs ranging from Genentech, Recursion Pharmaceuticals and Evozyne, among others. 

The momentum is continuing in early 2024, with NVIDIA announcing renewed pacts with Amgen and DeepCell. Amgen will use NVIDIA’s drug discovery generative AI platform BioNeMo Cloud service to accelerate molecule screening and optimization processes. Meanwhile, NVIDIA’s partnership with Deepcell hinges on advancing cell morphology studies with generative AI. “There is a very big moment happening in the world of pharmaceuticals and how drugs are being discovered,” said Kimberly Powell, vice president of Healthcare at NVIDIA, in a call with journali…

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Medable: AI-powered platform can halve trial build times

Decentralized clinical trial pioneer Medable unveiled an intelligent automation technology at the JP Morgan Health Care Conference, claiming it can slash standard trial build timelines by at least 50%. This development hinges around electronic clinical outcomes assessment (eCOA) — a common bottleneck in trial startups.

“We’ve used AI to essentially take a protocol and translate that into a mobile application that is running on a patient’s phone,” said Dr. Michelle Longmire, CEO of the company in a recent interview. “The app can partner with patients in their health journey, asking them the right questions at the right times to understand how they’re doing with their clinical trial intervention.”

The AI in question involves the use of large language models for complex task automation and workflow orchestrations.

Medable notes that prominent Big Pharma companies are using the automation technology to cut weeks off build times. “AI has sol…

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The global biotech funding landscape in 2023: U.S. leads while Europe and China make strides

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In 2023, the U.S. continued to demonstrate its position as the biotech funding leader, commanding over one-third, 35%, of the global investment in the sector. Overall, U.S. biotech firms attracted $56.79 billion in funding, according to a survey of Crunchbase data. Next in line was China, which contributed about 12.7% to the global funding pool, or $20.61 billion. Up next was Europe, which secured more than $11.46 billion and representing more than 7% of the worldwide funding.

While U.S. leads in total biotech funding, Chinese biotech companies, on average, saw larger funding rounds than either Europe or the U.S. The average funding size per company in China was roughly three times larger than that in the U.S. and six times larger than the average in Europe.

But while China-based companies had larger hauls, they were comparatively few. Chinese biotech secured …

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