2022 was a transformative year for biotechnology and 2023 is proving to be even more groundbreaking. Several key developments over the past year have demonstrated the incredible potential of biotech to treat previously incurable diseases, enhance human health, and positively impact the planet.
Gene Editing Takes Major Steps Towards Clinical Use
One of the most promising biotech advances has been in the field of gene editing. Powerful new gene editing technologies like CRISPR have continued rapid development towards real-world medical applications.
In November 2022, Intellia Therapeutics and Regeneron Pharmaceuticals announced positive interim results from an ongoing phase 1 clinical trial assessing their CRISPR-based therapy, NTLA-2001, for treating transthyretin (ATTR) amyloidosis. This rare, progressive condition causes buildup of abnormal protein deposits throughout the body and the trial results showed NTLA-2001 was safe and effective at lowering the disease-causing protein by 87% after a single infusion.
If efficacy is maintained in further trials, it could offer a one-time curative treatment for ATTR patients compared to current lifelong managements. The CEO of Intellia called it a “gamechanger”, while Regeneron said it has “set the stage for developing a new class of medicines.”
This trial marks one of the first major steps towards translating CRISPR from a laboratory technology into practical gene editing medicines. Its success has bolstered confidence that gene editing may soon be ready for mainstream approval and rollout.
In July 2023, Intellia and Regeneron plan to share 6 and 12 month follow-up results from their ATTR trial. If longer term safety and efficacy is demonstrated, they could file for regulatory approval in 2024 – an extremely accelerated timeline for such transformative medicines.
Gene editing advancements haven’t been without setbacks though. In October 2022, Cellectis released disappointing results from their phase 1 BALLI-01 study trialling UCART22 – an off-the-shelf CAR T therapy for acute lymphoblastic leukemia (ALL). It failed to show any meaningful cancer eliminating activity, causing their share price to drop 35% virtually overnight.
While this dealt a blow to ambitions around allogeneic CAR T treatments, Cellectis maintains this was likely due to suboptimal lymphodepletion regimens failing to properly prepare patient’s immune systems. They will continue to optimize and improve their approach through further R&D.
Overall though, gene editing and cell therapy has made giant leaps towards medical utility over the past year. With waters now being tested in human trials, it likely won’t be long before these revolutionary technologies transition into practical real-world treatments.
Obesity and Weight Management Innovation Accelerates
2023 has seen major developments around pharmaceutical treatments for obesity and weight management. With over 70% of American adults overweight, there is tremendous demand for medical solutions.
In September 2022, the FDA approved Wegovy – a weekly injection from Novo Nordisk that suppresses appetite through mimicking the hunger-regulating hormone GLP-1. Wegovy demonstrated average weight loss of 15% in clinical trials and has been hailed as a “gamechanger” for obesity medicine.
However, tremendous demand for Wegovy has led to extreme shortages, with Novo Nordisk failing to manufacture enough to meet market appetite. To combat waitlists now exceeding 1 million patients, the FDA just approved Ozempic – a similar anti-obesity injection from Novo Nordisk – for the same obesity and weight management indications.
| Drug | Manufacturer | Mechanism | Average Weight Loss |
|---|---|---|---|
| Wegovy | Novo Nordisk | GLP-1 Mimic | 15% |
| Ozempic | Novo Nordisk | GLP-1 Mimic | 12.4% |
This regulatory move will help expand supply of weight-loss injections to meet surging patient demand in 2023. However, even with extra manufacturing capacity from Ozempic, shortfalls are expected to persist through much of 2024.
Beyond pharma injections, 2023 is also seeing new medical devices enter the obesity treatment paradigm. In June 2022, the FDA approved an ingestible “Pacemaker for your stomach” called Barostim – developed by CVRx Inc. This implantable neuromodulation system stimulates the body’s cardiovascular system to improve metabolism and suppress appetite.
Early real-world usage of Barostim has demonstrated average excess weight loss of 21.2% at 5 months. While still early, some experts hail Barostim as “the most promising advance in treating obesity in decades”. If results remain durable long-term, implantable technologies like Barostim promise to be a powerful future treatment paradigm for obesity.
Between new medications, accelerating devices, and growing patient demand, the obesity treatment market is undergoing massive transformation. 2023 is likely to see continued innovation and expansion in this underserved disease space.
Gene Sequencing & AI Unlocks Mysteries of Long COVID
One silver lining that has emerged from the devastating COVID-19 pandemic is that it has massively accelerated research into complex, heterogeneous diseases. Nowhere has this been truer than for post-COVID condition, also known as “Long COVID” (LC).
While first described in 2020, LC has confounded researchers due to the sheer diversity of its over 200 reported symptoms and unknown root biological causes. However, over the past year, advanced technologies have started unlocking LC’s complex mysteries.
Genetic analysis of LC patients has identified associations with irregularities across 150 regions of the human genome. Gene locations tied to LC risk were involved in broad biological processes like metabolic inflammation pathways and antiviral responses.
Researchers have posited this supports theories of LC arising due to immune system dysregulation after viral encounters. Ongoing efforts cataloging the genetics of LC could enable future diagnostics predicting LC vulnerability and targeted treatments correcting identified irregularities.
Similarly, artificial intelligence (AI) analysis has identified distinguishable patterns among LC patients. Researchers from Mount Sinai health system built an AI algorithm assessing medical records – including doctor’s notes, test results, medications and procedures – from LC patients versus healthy controls. Their model was able to differentiate LC cases from controls with over 90% accuracy.
The AI was even able to identify distinctive multi-symptom phenotype “subgroups” of LC patients. For example, it revealed clusters with primarily respiratory symptoms, mental health disturbances, or cardiovascular complaints.
These AI-identified patient stratifications could enable tailored diagnostics, subgroup-specific treatments, and procuring well-defined participants for clinical trials. Researchers called it “a new path for precision medicine in long COVID-19.”
Combining the powers of gene sequencing and AI has rapidly progressed understanding of this complex, highly variable post-viral disease. While much work remains, researchers are optimistic that 2023 could see the first LC tailored diagnostics and targeted treatment options emerge.
Conclusion: Biotech Momentum Will Continue Building
Major leaps have clearly been made across gene editing, obesity medicine, and post-viral illness over the past year. These examples showcase how biotechnology’s tremendous potential is beginning to translate into tangible real-world impacts.
Trends like aging demographics, rising chronic illnesses, climate change vulnerabilities and now post-pandemic syndromes are escalating demand for biotech innovation. Luckily, accelerating technologies like CRISPR, RNA therapeutics, AI-driven R&D seem poised to rise to the growing challenges ahead.
Judging by the immense progress made in 2022 alone, the biotechnology transformation has only just begun. 2023 is likely to build further momentum towards unlocking biotech’s immense promise for radically advancing human health and flourishing on this planet.
To err is human, but AI does it too. Whilst factual data is used in the production of these articles, the content is written entirely by AI. Double check any facts you intend to rely on with another source.
