January 15, 2024
The biopharma sector began a turbulent year with several upheavals in 2023, mirroring trends seen in many industries. Both large and small biotech companies experienced extensive layoffs and notable changes in leadership.
For startups, there’s been a notable strategic shift among investors. A recent Pitchbook report indicates a significant reduction in activity this year, with approximately 840 deals amounting to around $24 billion. This marks a steep decline from the over 1,500 deals totaling nearly $60 billion in 2021. A key factor in this downturn is the spring collapse of Silicon Valley Bank, which led to funding difficulties and heightened investment wariness.
The report also highlights a strategic pivot in deal-making in 2023, observing a trend towards fewer, yet more substantial deals. Venture capitalists are now placing greater emphasis on startups with solid clinical outcomes and biotech firms dedicated to platform development for multiple drug creations, rather than companies focused on one or two therapies.
Despite these challenges, 2023 witnessed significant technological breakthroughs in biotech, alongside shifts in investment approaches.
Here are three pivotal moments and trends from 2023 that have significantly influenced biotech and will continue to shape its future.
FDA Greenlights First Crispr Gene Therapy
In January 2013, groundbreaking research introduced the world to “clustered regularly interspaced short palindromic repeats,” better known as Crispr, a method for gene editing using bacterial genome sequences. These studies showcased the potential of these bacterial mechanisms to streamline the correction of genetic disorders. Fast forward over ten years to December 2023, and this potential turned into reality with the FDA’s approval of the first Crispr-based gene therapy.
Vertex Pharmaceuticals, in collaboration with Crispr Therapeutics—a company co-founded by Nobel Prize laureate Emmanuelle Charpentier for her work in Crispr—developed this revolutionary drug, named Casvegy. It offers a new treatment for sickle cell anaemia, involving the extraction, genetic modification, and reinsertion of bone marrow cells in patients. Vertex’s data, revealed shortly after the drug’s approval, confirmed that the genetic alterations were sustained four years post-treatment.
This marks just the start of Crispr technology’s journey in gene therapy. Already, the clinical pipeline is brimming with dozens of Crispr-based drugs. Since its initial discovery, scientists have been honing next-generation Crispr tools for more precise gene editing, even exploring the possibility of in vivo gene modification. The biotech industry has seen a surge of companies leveraging these advanced technologies, with the first drugs developed using these next-generation tools entering clinical trials.
Whole Genome Sequencing Now Available in IVF Clinics
Following the completion of the Human Genome Project in 2003, genome sequencing has undergone remarkable advancements, becoming more efficient, rapid, and applicable. Traditional sequencing methods involved breaking DNA into small fragments for quick analysis and arrangement, yielding substantial breakthroughs but also limiting the depth of genetic insights obtainable.
However, the recent introduction of “whole genome” sequencing has revolutionised this process. This approach involves cutting DNA into larger segments, simplifying the task for sequencing machines to accurately reassemble them. This method not only clarifies the structure of an individual’s DNA but also enhances understanding of gene functionality within the body.
In 2023, whole genome sequencing began transitioning from research and public health settings to practical clinical use. This month, Orchid, a company supported by 23andMe Co-founder Anne Wojcicki and Coinbase CEO Brian Armstrong, declared the availability of its whole genome sequencing for embryos in IVF clinics. This service allows prospective parents to assess embryos for genetic risks, potentially increasing the likelihood of a successful pregnancy.
Children’s Mercy Hospital in Kansas City made a significant announcement in October. The hospital, utilising sequencers from PacBio, started employing whole genome sequencing in clinical settings. This advancement will speed up the hospital’s ability to diagnose genetic disorders, as explained by Tomi Pastinen, the hospital’s genomic medicine director. Pastinen expressed his hope that this move by his hospital will encourage similar adoption elsewhere, stating, “We hope that clinical adoption is the catalyst to really open the floodgates for these types of genome sequencing.”
A Startup’s Innovative Approach to Prolonging Canine Lifespan
The quest to discover the secret of extending healthy human lifespans has led to an abundance of books, supplements, and podcasts, yet there remains no officially sanctioned medication that can prolong the life of humans or any other species. This is the gap Celine Halioua’s company, Loyal, aims to fill by developing drugs that could potentially increase the lifespan of dogs, and perhaps eventually, humans.
In the recent spring, the FDA greenlit the first clinical trials for LOY-001, Loyal’s pioneering drug intended to extend the lives of large, older dogs by reducing the hormone responsible for their size. Following successful preliminary studies in the fall, the company received critical endorsement from the FDA. The data presented was convincing enough to suggest that LOY-001 could effectively increase the lifespan of larger dogs.
Loyal’s immediate plan is to secure the remaining FDA approvals necessary to commercialise LOY-001. Beyond this, Halioua envisions applying insights gained from canine longevity research to develop preventive treatments for age-related diseases in humans.
Empowering Biotech Breakthroughs
Reflecting on this transformative year in biopharma, marked by advances in gene therapy, genome sequencing, and lifespan extension research, we recognise the vital importance of leadership in these groundbreaking areas. As the industry evolves, our expertise in connecting visionary leaders with pioneering biotech companies becomes increasingly crucial. Our role is not just to observe but to actively participate in this evolution, ensuring that the firms at the forefront of these breakthroughs are guided by the most capable and innovative minds, ready to shape the future of biotechnology.
Published on 15-01-2024