2023 was quite year, to say the least! Among the things that we witnessed: The second biggest bank collapse in US history, layoffs across biotech, AI/ChatGPT dominated the scene, AlphaFold received the Lasker price, discoveries of nucleoside base modifications that enabled the development of effective mRNA vaccines against COVID-19 won the Noble Prize, Science magazine names GLP-1 receptor agonists (think Ozempic) as the “2023 breakthrough of the year”, FDA approved first gene therapy for treatment of Duchenne Muscular Dystrophy and first CRISPR gene editing treatment for sickle cell disease, and there is more….
AI/ChatGPT everywhere
AI must be regulated
AI dominated the scene with ChatGPT – it only broke publicly into the world at end of 2022 – and healthcare teach leaders are already eager to adopt the technology sooner than later and as a consequence, striking partnerships are being formed (e.g., Amazon with 3M to advance conversational AI in medicine, Microsoft and Epic to bring AI into electronic health records). While recognized by the FDA commissioner Robert Califf that large language models (LLMs), such as ChatGPT, will result in important healthcare applications, he also made it clear that ChatGPT-like models require regulation to avoid being swept up quickly by tech as the potential danger lies in uncontrolled applications and the future upkeep. OpenAI CEO, Sam Altman, faced questions about Artificial Intelligence at a hearing in the U.S. Senate with the tech executive and lawmakers agreed that new AI systems must be regulated. Interestingly, shortly thereafter OpenAI had its share of prominent and not so favorable visibility with closing a $300 million funding round at $27billion-$29 billion valuation, and nearly imploding after CEO Sam Altman was fired and then being rehired again.
AlphaFold Received the Albert Lasker Medical Research Award
The incredible advancements in the AI field continued with Google DeepMind classifying the potential effects of 71 million ‘missense’ mutations to pinpoint the causes of genetic disease, made public via the release of AlphaMissense (a catalogue of ‘missense’ mutations) which summarizes what possible effect each of these mutations may have. This feat followed Google DeepMind’s release of the AlphaFold Protein Structure Database (AlphaFold DB) two years earlier, which by now includes the predicted structure of nearly 200 million catalogued proteins. To no one’s big surprise, the 2023 Albert Lasker Basic Medical Research Award, announced during the month of September, now honors the two scientists (Demis Hassabis and John Jumper) who invented AlphaFold.
Commercial shakeups and the second biggest bank collapse in U.S. history
The year also saw some commercial shakeup across the healthcare/precision medicine industry with numerous layoffs, which started end of 2022 and included restructurings to cut costs mostly due to difficult operating environments. The result were 119 layoff events in the biotech industry alone as summarized in the Fierce Biotech Layoff tracker (compared to 23 in 2023). Clearly, after years of easy money and heady growth, funding has slimmed and stocks are down. Companies are said to be waiting for the J.P. Morgan Healthcare Conference, taking place in January 2024, to see whether and how the industry is going to reset in the short term.
A precedent – or a parallel – of all of these shakeups included the second biggest bank collapse in U.S. history when the Silicon Valley Bank was shut down by regulators in March. The company’s downward spiral accelerated dramatically when surprised investors learned that the bank needed to raise $2.25 billion to shore up its balance sheet. The Silicon Valley Bank, a 40-year-old institution, had been one of the most prominent lenders in the world of technology startups. The U.S. Treasury, the Federal Reserve, and the Federal Deposit Insurance Corp. eventually came to the rescue which ended up saving a lot of start-ups including companies in the healthcare sector.
Precision Medicine builds upon the previous year’s gains
While the above mentioned news rattled the markets and the industry in general, the precision medicine sector has been nicely and steadily building upon the previous year’s achievements with significant technological advancements and opportunities for medicine being reported. And as a result, 2023 was not short of anything with companies reaching milestones, major acquisitions or announcements having occurred, significant technologies being launched, and research findings being published.
- Recent FDA approvals (FDA approved first gene therapy for treatment of certain patients with Duchenne Muscular Dystrophy and first CRISPR gene editing treatment for sickle cell disease) highlight and illustrate how technological advancements and a greater understanding of the underlying mechanisms of disease can lead to targeted/personalized treatment.
- Some of the highlights of genetics and medicine that shaped 2023 include Illumina started to ship the NovaSeqX Plus sequencer (which provides a $200 USD genome), Illumina launched its first product which enables long-read and short-read sequencing on one instrument, and this while PacBio began the commercialization of their Onso short-read sequencing system. Velsera was formed as a result of combining Seven Bridges with PierianDx and Urgentec, the American College of Medical Genetics (ACMG) updated its guidance for reporting secondary findings in the context of clinical exome and genome sequencing (v3.2), the invention of a new way to map specific DNA markings called 5-methylcytosine(5mC) was reported which regulate gene expression and thus play a key role in health and disease and which potentially can be used in liquid biopsy applications the, Broad Institute launched the $1K Sample-to-Report Clinical Whole-Genome Sequencing Service, and genome sequencing was being shown to be highly effective at diagnosing genetic disorders in newborns and infants. Lastly, in October Katalin Karikó and Drew Weissman received this year’s Nobel Prize in Physiology or Medicine for their discoveries of nucleoside base modifications that – among other things – enabled the development of effective mRNA vaccines against COVID-19.
- Research findings were substantial and varied – to name a few: In August the completion of the full sequence of the Y chromosome was published in two different papers, the NIH unveiled a comprehensive dataset to help cancer researchers uncover new molecular insights into how cancers develop and progress that standardizes genomic, proteomic, imaging, and clinical data from individual studies of more than 1,000 tumors across 10 cancer types, and scientists revealed how proteins drive growth of multiple cancer types. Furthermore, it was shown that centenarians have a diverse gut virome with the potential to modulate metabolism and promote healthy lifespan, organ aging signatures in the plasma proteome that track health and disease were identified, and the rich multi-omics pan-cancer proteomic dataset was released, connecting genomic aberrations to cancer phenotypes. Lastly, the genomic analyses of eight locks of hair attributed to Ludwig van Beethoven shedding light on his chronic health problems and cause of death – Beethoven had a predisposition for liver disease and became infected with hepatitis B.
New weight loss drugs – 2023 breakthrough of the year
Science magazine declared the new drug class of GLP-1 receptor agonists as the breakthrough of the year 2023. Originally developed for treatment of diabetes, these medicines are now breaking the mold, and there is a groundswell of hope that they may dent rates of obesity and interlinked chronic diseases. It all started about two years ago, when Novo Nordisk’s next iteration, semaglutide, was greenlit for weight management (marketed as Ozempic for diabetes and Wegovy for obesity). Following the success of Ozempic, the FDA has now (December 2023) also approved Eli Lilly’s diabetes drug Zepbound – another GLP-1 receptor agonist – for chronic weight management.
Illumina/GRAiL-specific timelines
- In June: the resignation of Francis deSouza as CEO of Illumina after a proxy battle with Icahn in relation to the controversial $7.1 billion acquisition of GRAiL. Jacob Thaysen has since (in September) taken on the role of CEO at Illumina.
- In July: Illumina was hit with a record $476M EU antitrust fine over the GRAiL deal which followed the layoff news announced in the prior month as part of an effort to cut more than $100M in expenses in 2023.
- In August: Illumina was being investigated by the SEC in relation to the $7.1B Grail acquisition, because they completed the merger in August 2021 before the Commission had weighed in on whether the deal was anti-competitive.
- In September: Illumina being ordered by EU regulators to sell the cancer test developer GRAiL.
- In December: Illumina sets a date to divest GRAiL following antitrust appeal ruling. Illumina will look to start fresh as it sets a timeline to separate itself fromGrail and its cancer-detecting blood test.
And let’s not forget
PerkinElmer rebranded its diagnostics, life sciences business as Revvity, Colossal Biosciences, the world’s first de-extinction company, was named to the third-annual TIME100 Most Influential Companies list, Myriad Genetics announced a new $90M asset-based credit facility, Amazon launched a generative AI-based clinical documentation service, the Genomics team at Google Health released the new method DeepSomatic which detects subclonal variants from tumor and normal input BAMs, PacBio announced a complete computational workflow for human whole genome sequencing data analysis, and Genomics England announced a list of rare conditions that will be included in the Generation Study which will look at the DNA of over 100,000 babies to consider whether whole genome sequencing could be rolled out as part of a future newborn screening program,
Lastly, in 2023 major acquisitions happened (CVS Health completed the purchase of Medicare-focused primary care provider Oak Street Health in an all-cash deal valued at $10.6 billion, Kaiser Permanente acquired Geisinger Health, Thermo Fisher Scientific acquired Olink, Roche entered into a definitive agreement to acquire LuminarDx’s point of care technology, Amazon completed its One Medical acquisition for $3.9 billion, and PacBio entered into an agreement to acquire Apton Biosystems to accelerate the development of a next generation, high-throughput, short-read Sequencer) and substantial investments were made (Clinical Trial company Paradigm launched with $203M in funding, Causaly raised $60M to catalyze their AI-powered preclinical discovery platform, Harbinger Health raised $140M to accelerate advancement of its screening platform for early-stage cancer, AI-focused startup Generate:Biosciences raised $273M, and Viome Life Sciences closed $86.5M), this the while Health AI startup Olive, once valued at $4 billion, shut down.
Brigitte Ganter, PhD – enlightenbio Guest Blogger
Brigitte Ganter, is currently Sr. Director of Product Marketing at L7 Informatics. Prior to her work at L7, Brigitte had founded enlightenbio LLC where she oversaw as General Manager all aspects of the company’s activities with particular focus on product management and product marketing services, including market research reports. Brigitte holds a PhD from the Swiss Federal Institute of Technology, Zurich (ETH Zurich) and conducted her post-doctoral work at Stanford University. Her broad industry experience includes positions of increasing responsibility predominantly in the role of Director of Product Management at several biotechnology/technology startups (Iconix Biosciences, Ingenuity System [now a QIAGEN company], DNAnexus).
