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2023-12-27| Special

GeneOnline’s Pick: Top 10 Global Biotech News Stories in 2023

by Sinead Huang
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In 2023, a new chapter begins with the end of the COVID-19 pandemic. On May 5th, Dr. Tedros Adhanom Ghebreyesus, Director-General of the World Health Organization, declared that the COVID-19 pandemic no longer poses a public health emergency of international concern. Other than the impact of the COVID-19 pandemic, several notable events have stirred the global biotech and pharmaceutical industry. As the end of 2023 approaches, explore GeneOnline’s overview of breakthrough research and cutting-edge technology throughout the year.

1. The Potential of mRNA Vaccine

The rapid emergence of mRNA vaccines during the COVID-19 epidemic has been notable, with mRNA-1273 and BNT162b2 standing out for their high efficacy demonstrated in clinical trials. These vaccines, which have also been approved for use against SARS-CoV-2 variants through bivalent formulations, showcase not only effectiveness but also safety.

Beyond their success in COVID-19, mRNA vaccines show considerable promise in addressing other infectious diseases such as Zika virus and influenza. The exploration of mRNA vaccines extends to cancer therapeutics, where they target tumor-associated antigens or neoantigens, with ongoing clinical trials covering a spectrum of cancer types. Despite these advancements, the application of mRNA vaccines in immunological diseases, tissue damage, and rare diseases is still in the early stages of investigation. This nascent exploration underscores the existing gaps in knowledge regarding the efficacy and challenges associated with mRNA vaccines in these diverse medical domains.

2. Pharmaceutical Giants Shift Focus to RSV Prevention Amid COVID Vaccine Decline

The vaccine industry’s current focus has shifted to preventing the Respiratory Syncytial Virus (RSV) in the wake of declining demand for COVID-19 vaccines. Major pharmaceutical companies such as GSK, Pfizer, Moderna, and AstraZeneca are actively developing RSV vaccines, eyeing a market estimated to be worth over $5 billion and projected to exceed $10 billion by 2030.

RSV, a common respiratory virus, poses significant health risks, particularly for infants and older adults. With approximately 64 million infections and 160,000 deaths annually, the economic burden on healthcare systems is substantial. Recent developments include GSK being the first to receive FDA approval for Arexvy, targeting older adults. Pfizer’s Abrysvo also gained approval, with Pfizer aiming to lead in the maternity and pediatric market. 

Click here to read more about the RSV vaccine.

3. Revolutionizing Medical Applications in AI

GPT-4, the latest model developed by OpenAI, represents a significant breakthrough in AI development, designed to acquire general cognitive intelligence. The model exhibits enhanced comprehension, reasoning, and response capabilities, mimicking human-like intelligence.

In the medical field, GPT-4 demonstrates potential applications such as medical examinations, patient care, and clinical documentation. It has been tested in scenarios like the United States Medical Licensing Examination (USMLE), showcasing its ability to provide accurate responses and reasoning. GPT-4’s proficiency extends to curbside consultations, offering accurate answers in 93% of cases. Notably, in a study comparing responses from healthcare professionals and GPT-4, the AI was preferred 79% of the time for its quality and empathy. Despite some limitations, such as hallucinations and challenges in math and logic, GPT-4’s role in automating medical tasks and streamlining processes, including medical education, highlights its potential impact in the healthcare sector. 

To read more about GPT-4, please visit here.

4. Gut Microbiota Composition Unveils Early Alzheimer’s

Gut microbiota composition has been one of  the main topics in 2023. In a recent study published in Science Translational Medicine, researchers from Washington University School of Medicine in St. Louis explored the link between gut microbiome composition and Alzheimer’s disease (AD).

Examining individuals in the preclinical stage of AD, the researchers identified significant differences in gut microbiota between this group and cognitively normal individuals, even with similar diets. Importantly, the team demonstrated that integrating these microbiome features into machine learning algorithms improved the prediction of preclinical AD status. The findings suggest the potential for utilizing gut microbiota as biomarkers for early AD detection, enabling simpler and less invasive screening methods compared to traditional approaches like brain scans or spinal taps. 

The study opens avenues for future research into causal links between the gut microbiome and AD, paving the way for interventions and treatments aimed at altering the microbiome to potentially slow down or prevent the development of AD symptoms. However, the researchers emphasize the need for further validation and exploration of causality in larger populations.

Click here to learn more about how gut microbiome composition offers insight for Alzheimer’s.

5. A Landmark in CRISPR-Based Gene Therapy

After being awarded the Nobel Prize in 2020, CRISPR technology has been attracting public attention. Pharmaceutical companies also hope to use this technology to develop drugs that help people. CASGEVY, the first FDA-approved treatment utilizing CRISPR genome-editing technology, received approval on December 8, 2023, marking a groundbreaking achievement in gene therapy. 

Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, CASGEVY offers hope to individuals with severe sickle cell disease (SCD). Targeting autologous CD34+ hematopoietic stem cells, CASGEVY leverages CRISPR/Cas9 to edit the genome and reduce BCL11A expression, leading to increased fetal hemoglobin production. This innovative approach aims to alleviate vaso-occlusive crises, significantly improving the quality of life for the approximately 16,000 eligible patients aged 12 and older with recurrent Vaso-Occlusive Crisis (VOC). The companies are collaborating with top U.S. hospitals to establish Authorized Treatment Centers (ATCs) for the administration of CASGEVY, with plans to expand accessibility in the coming weeks. The approval represents a milestone in SCD treatment and advances the frontier of CRISPR-based therapies, offering potential therapeutic avenues for patients in need.

Continue reading news about CASGEVY here.

6. Revolutionizing Type 2 Diabetes Treatment with Innovative Drug Delivery

Diabetes, a chronic disease resulting from insufficient insulin production or ineffective use, leads to hyperglycemia and significant damage to the body’s systems, contributing to global mortality. Stanford University researchers, in collaboration with Novo Nordisk, have developed a drug delivery system utilizing an injectable biomimetic hydrogel carrier combined with GLP-1 receptor agonists, commonly prescribed for weight loss and blood glucose regulation. 

The innovative approach, tested on diabetic rats, extends drug action to approximately six weeks, potentially revolutionizing type 2 diabetes treatment. By reducing the frequency of injections to once every four months, the system addresses challenges of patient adherence, a critical issue in managing diabetes. With the global diabetic population expected to exceed 1.3 billion by 2050, this advancement presents a promising solution to improve patient compliance, potentially reducing healthcare expenditure related to diabetes. The polymer-nanoparticle hydrogel, easily injectable and stable for up to four months, shows efficacy comparable to daily dosing, paving the way for human clinical trials in the near future.

To learn more about the innovative drug delivery system, click here.

7. A Breakthrough in Treating Chronic Retinal Diseases

A research team from the University of California, Irvine (UCI) School of Medicine has identified a novel group of small-molecule drugs, known as “stress resilience-enhancing drugs” (SREDs), with potential clinical utility in treating blindness caused by chronic retinal diseases such as age-related macular degeneration (AMD), diabetic retinopathy (DR), and retinitis pigmentosa (RP). 

With limited therapeutic options available for these progressive diseases, the study targeted an unmet medical need, employing a systems pharmacology platform to identify universal molecular mechanisms. The SRED therapeutic intervention demonstrated enhanced resilience to acute and chronic forms of stress in degenerating retinas, preserving tissue structure and function across various retinal disease models. This innovative class of drugs offers hope for treating or preventing the most common causes of blindness and may become a standard of care, providing new possibilities for patients in earlier stages of disease progression.

Read the news here.

8. Micronuclei’s Role in Cancer Metastasis Mechanisms

Chromosomal instability (CI) and epigenetic alterations are identified as two major indicators for cancer cell metastasis. Albert Agustinus, a graduate student from the Memorial Sloan Kettering Cancer Center (MSK), discovered that micronuclei (MCN) generated due to chromosomal instability accumulate significant epigenetic changes during cell division, making cancer more challenging to treat. Published in the journal Nature, this study not only opens up a new and promising research direction in basic science but also holds potential applications in clinical care. The research suggests that the relationship between micronuclei and chromosomal instability could offer new insights into more effective cancer treatments and prevention of cancer metastasis.

9. Synthetic Embryos Created without Eggs and Sperm

Scientists in the United States and the United Kingdom claim to have developed the world’s first synthetic human embryo-like structures using stem cells, bypassing the traditional need for eggs and sperm. Although in the earliest stages of human development without a beating heart or brain, these synthetic embryo models could potentially advance understanding of genetic diseases and miscarriage causes. The lack of clear regulations for stem cell-derived models of human embryos raises legal and ethical questions, and experts call for urgent regulations to guide their creation and use. Researchers aim to unravel the mysteries of human development, focusing on the critical period after 14 days post-fertilization, which is the current limit for studying embryos in labs. While these synthetic embryos are currently confined to test tubes, scientists hope they will provide insights into preventing pregnancy loss.

10. First-Ever Whole-Eye and Face Transplant Success

A surgical team at NYU Langone Health achieved a medical milestone by conducting the world’s first whole-eye and partial-face transplant on a 46-year-old military veteran who survived a high-voltage electrical accident. The procedure involved transplanting the entire left eye and part of the face from a single donor, marking the first-ever human whole-eye transplant. While the patient’s vision restoration remains uncertain, the transplanted eye has exhibited remarkable signs of health, including direct blood flow to the retina. This unprecedented achievement not only opens new avenues for advancements in vision therapies but also prompts discussions on the ethical and legal aspects of such complex medical procedures. The success of the surgery, performed by a multidisciplinary team, paves the way for future innovations in transplantation and related fields, offering hope to individuals with severe facial and ocular injuries.

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