Search Results

< Back Who Will Win the 2024 Nobel Prize in Physiology or Medicine? As we approach the 2024 Nobel Prize announcement, we highlight three potential candidates whose research promises to reshape the future of healthcare. The Nobel Prize in Physiology or Medicine is one of the most prestigious honors in science, celebrating breakthroughs that significantly advance human knowledge and improve lives. Established by Alfred Nobel’s will, the prize has recognized extraordinary achievements that have reshaped our understanding of biology and medicine. From the discovery of insulin to the development of mRNA vaccines , laureates have been those whose work has had a profound and lasting impact on global health. Notable figures such as Camillo Golgi, Santiago Ramón y Cajal, and Katalin Karikó have left a legacy through their contributions, continuing to influence medical research and treatment today. As we approach this year’s Nobel Prize announcements, scheduled from October 7 to 14, there is growing anticipation about which groundbreaking achievements will be honored. In this article, we highlight three exceptional researchers who have made substantial impacts on global health: Zhijian Chen, known for discovering the cGAS enzyme; Lotte Bjerre Knudsen, who revolutionized treatments for diabetes and obesity with GLP-1-based therapies; and Carl June, whose development of CAR T-cell therapy has transformed cancer treatment. These three stand out for their significant contributions, making them strong contenders for the 2024 Nobel Prize in Physiology or Medicine. Overview of the Nobel Prize Selection Process The Nobel Prize in Physiology or Medicine is awarded to up to three researchers whose work has profoundly influenced human health and medical science. The selection process is confidential and follows several steps. Each year, the Nobel Committee sends over 3,000 invitations to scientists, academics, and past laureates to submit nominations. These nominations are reviewed rigorously, leading to a shortlist, after which the committee consults experts in the relevant fields. Once the committee has made its recommendations, the Nobel Assembly at the Karolinska Institutet votes on the final laureates, a decision kept secret until the official announcement in early October. This high level of confidentiality, with nomination records sealed for 50 years, adds to the prestige and mystique of the Nobel Prize. This year, amid a wealth of potential nominees, three researchers—Chen, Knudsen, and June—stand out for their groundbreaking contributions in immunology, metabolic disorders, and cancer treatment. Zhijian "James" Chen, Ph.D.: Redefining Immunology Dr. Zhijian Chen's discovery of the cyclic GMP-AMP synthase (cGAS) enzyme is a revolutionary finding that has significantly deepened our understanding of the immune system’s response to foreign DNA. His work, which identified the cGAS-STING pathway, provides critical insights into how the body defends itself against viral infections, cancer, and even autoimmune diseases. Before Chen’s discovery, scientists lacked a clear explanation of how the immune system detected cytosolic DNA, especially from pathogens or damaged cells. Chen's breakthrough lies in the mechanism by which the cGAS enzyme recognizes foreign DNA in the cytoplasm—a signal that DNA is outside its normal place in the nucleus or mitochondria. When cGAS detects this DNA, it synthesizes cyclic GMP-AMP (cGAMP), a second messenger that binds to and activates the STING (Stimulator of Interferon Genes) protein. This activation triggers a cascade of immune responses, leading to the production of type I interferons and pro-inflammatory cytokines, which play a critical role in fighting infections and eliminating cancerous cells. Implications in Autoimmune Disease and Cancer One of the most significant aspects of Chen’s discovery is its dual role in fighting diseases. While the cGAS-STING pathway is essential for immune defense, its overactivation can lead to autoimmune diseases such as systemic lupus erythematosus (SLE), where the immune system mistakenly attacks healthy cells. Understanding this balance has allowed researchers to develop cGAS inhibitors that could prevent excessive immune responses in conditions like lupus and arthritis. Early-stage research has already shown promise in animal models, offering hope for new treatments for chronic inflammatory diseases. At the same time, the therapeutic potential of activating the cGAS-STING pathway in cancer immunotherapy is also being explored. STING agonists , which stimulate this pathway, are currently in clinical trials to enhance the immune system’s ability to detect and destroy cancer cells. When combined with other treatments such as immune checkpoint inhibitors, these drugs could significantly improve outcomes for cancer patients, making tumors more susceptible to immune system attacks. Ongoing Research and Recognition Dr. Chen’s discovery has opened the door to a new field of research, with pharmaceutical companies developing drugs that either inhibit or activate the cGAS-STING pathway, depending on the condition being treated. Researchers are also exploring this pathway’s role in infectious diseases, including tuberculosis and herpes, where boosting immune responses could lead to better treatments. In recognition of his groundbreaking work, Dr. Chen was awarded the 2024 Albert Lasker Basic Medical Research Award, a significant honor often considered a precursor to the Nobel Prize. His research has reshaped the field of immunology and continues to guide the development of therapies for a wide range of diseases, including autoimmune disorders, cancer, and infectious diseases. Lotte Bjerre Knudsen: Revolutionizing Metabolic Disease Treatment Lotte Bjerre Knudsen’s work on GLP-1-based therapies has transformed the treatment of two of the world’s most widespread chronic diseases: type 2 diabetes and obesity. As a pharmaceutical scientist at Novo Nordisk , Knudsen was instrumental in translating the hormone GLP-1 (glucagon-like peptide-1) into treatments that have dramatically improved the lives of millions of patients worldwide. GLP-1: The Science Behind the Treatment GLP-1 is a hormone that the gut releases in response to eating. It plays a vital role in regulating blood sugar by stimulating insulin production, inhibiting glucagon release, and slowing gastric emptying, which leads to a feeling of fullness. While GLP-1 was known to have potential therapeutic benefits, its natural form is rapidly degraded in the body, limiting its effectiveness. Knudsen and her team overcame this challenge by developing long-acting GLP-1 analogs, such as liraglutide and semaglutide, which can last much longer in the bloodstream. Liraglutide , which was first approved for the treatment of type 2 diabetes, later became a groundbreaking treatment for obesity. Semaglutide, a newer drug with a longer half-life, allows for weekly dosing and has shown even greater success in managing both blood sugar levels and inducing weight loss. In clinical trials, patients using semaglutide experienced weight loss averaging 15% of their body weight, an outcome that far surpasses previous obesity treatments. Expanding Beyond Diabetes and Obesity Beyond its impact on diabetes and obesity, GLP-1 receptor agonists have shown promising cardioprotective benefits, significantly reducing the risk of heart attack and stroke in patients with diabetes. This dual action on both metabolic and cardiovascular health has made GLP-1 therapies a cornerstone in treating these chronic conditions. Knudsen’s research is now influencing the treatment of other conditions linked to metabolic dysfunction, such as nonalcoholic steatohepatitis (NASH) and chronic kidney disease. Emerging studies also suggest that GLP-1 receptor agonists may have anti-inflammatory effects, opening new possibilities for treating inflammatory diseases like asthma and NASH, further broadening the therapeutic reach of these drugs. Clinical Impact and Recognition The impact of GLP-1-based therapies on patient outcomes has been nothing short of transformative. Both liraglutide and semaglutide have become essential tools in managing not only diabetes and obesity but also improving overall health outcomes. Recognizing her contributions, Knudsen was awarded the 2024 Lasker~DeBakey Clinical Medical Research Award. Her work continues to influence the future of chronic disease management, with potential applications extending well beyond metabolic disorders. Carl June, M.D.: Transforming Cancer Treatment with CAR T-Cell Therapy Dr. Carl June has revolutionized cancer treatment with his development of CAR T-cell therapy, a type of immunotherapy that has dramatically improved outcomes for patients with certain types of cancer. This innovative therapy uses the body’s immune system to target and destroy cancer cells, particularly in hematologic cancers such as acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma. The Science of CAR T-Cell Therapy CAR T-cell therapy involves collecting a patient’s T cells—a type of white blood cell that plays a key role in the immune response—and genetically modifying them to express chimeric antigen receptors (CARs). These receptors are engineered to recognize specific antigens on the surface of cancer cells. Once the modified T cells are re-infused into the patient, they multiply and actively seek out and destroy cancer cells. In clinical trials, CAR T-cell therapy has shown unprecedented success. For instance, in patients with relapsed or refractory ALL, complete remission rates as high as 83% have been observed. The first FDA-approved CAR T-cell therapy, Kymriah (tisagenlecleucel), has provided life-saving options for patients who previously had few or no effective treatments. Challenges and Future Applications Despite its success, CAR T-cell therapy is not without challenges. The therapy can cause severe side effects, including cytokine release syndrome (CRS) and CAR T-cell-related encephalopathy syndrome (CRES). CRS results from an overactivation of the immune system, while CRES affects the central nervous system. Managing these side effects has required the development of new protocols, including the use of tocilizumab to treat severe CRS. Dr. June and his team are now working on expanding the use of CAR T-cell therapy beyond hematologic cancers to treat solid tumors, which are more challenging due to the complex tumor microenvironment. Innovative approaches, such as armored CAR T cells that secrete immune-enhancing proteins, are being tested in early-stage clinical trials for cancers like pancreatic and ovarian cancer. Recognition and Global Impact For his pioneering work, Dr. June received the 2024 Breakthrough Prize in Life Sciences, one of the most prestigious awards in science. His contributions have not only saved countless lives but also laid the foundation for future breakthroughs in cancer immunotherapy. As research continues, CAR T-cell therapy has the potential to become a more widespread treatment for both blood and solid tumors, offering hope to even more patients in the years to come. A New Era in Medicine: Conclusion and Broader Implications Zhijian Chen, Lotte Bjerre Knudsen, and Carl June represent three of the most important scientific advancements in recent history. Despite their work spanning diverse areas—immunology, metabolic disease, and cancer therapy—all three researchers have fundamentally changed how we approach disease treatment. Their contributions mark a shift towards precision medicine, where treatments are increasingly personalized and tailored to specific biological mechanisms. Dr. Chen’s discovery of the cGAS-STING pathway has provided new opportunities to treat autoimmune diseases, cancer, and infections by fine-tuning the immune response. Knudsen’s GLP-1-based therapies have not only transformed diabetes and obesity treatment but also paved the way for future therapies targeting related metabolic disorders and chronic conditions. Carl June’s CAR T-cell therapy has redefined cancer treatment, particularly for patients with previously untreatable blood cancers, and holds promise for solid tumors. It’s important to note that this is just our take on who might be among the strongest contenders for the 2024 Nobel Prize in Physiology or Medicine. The field of biomedical research is rich with talent, and there are countless other researchers whose extraordinary work deserves recognition. No matter who is ultimately awarded the prize, it’s certain that their contributions to science and healthcare will be profound and deserving of this prestigious honor. The global impact of the work by these three scientists is clear. From reducing the burden of chronic diseases like diabetes and obesity to offering life-saving cancer treatments, their breakthroughs are already improving healthcare outcomes for millions of patients. As their therapies continue to evolve, the potential to scale these treatments for broader access promises even greater global health benefits. Their collective achievements underscore the ripple effect of innovation in science: each discovery builds on existing knowledge, opening new pathways for research and treatment. Whether targeting the immune system, metabolic dysfunction, or cancer cells, the work of Chen, Knudsen, and June showcases the power of modern medical science to address some of the most complex challenges in healthcare. As we await the 2024 Nobel Prize announcements, the contributions of these researchers highlight a future where precision, personalized treatments will dominate the medical landscape, providing more effective and individualized care for patients across the globe. Whoever the winners may be, their contributions to medicine and science will be a testament to the brilliance of today’s researchers. Stay tuned—these innovations represent just the beginning of what's possible in the rapidly evolving field of medical science. Read our breakdown of the science behind the 2023 Nobel Prize for Medicine winners here . Author Ramya Nadig , freelance contributor Previous Next

< Back World Health Summit 11th - 13th October, 2026 Berlin, Germany From Crisis to Resilience: Innovating for Health. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next

A groundbreaking clinical trial set to begin in summer 2026 will test LungVax, the world’s first vaccine designed to prevent lung cancer in people at high risk, a major milestone in cancer prevention research. < Back World-First Preventative Lung Cancer Vaccine Enters Clinical Trial A groundbreaking clinical trial set to begin in summer 2026 will test LungVax, the world’s first vaccine designed to prevent lung cancer in people at high risk, a major milestone in cancer prevention research. Developed by researchers at the University of Oxford and University College London, and backed by Cancer Research UK and the CRIS Cancer Foundation, LungVax aims to prime the immune system to detect and destroy abnormal lung cells before they turn cancerous. “Lung cancer is lethal and blights far too many lives. Survival has been stubbornly poor for decades. LungVax is our chance to do something to actively prevent this disease,” said Professor Sarah Blagden, co-founder of the LungVax project. How LungVax Works LungVax uses a viral vector technology based on the same platform that powered the Oxford/AstraZeneca COVID-19 vaccine. It delivers genetic instructions to the immune system so it can recognise “red-flag” proteins (neoantigens) on the surface of early abnormal lung cells, marking them for the immune system to destroy. The Phase I trial will focus on individuals at particularly high risk, including patients who have had early-stage non-small cell lung cancer removed, as well as people currently enrolled in NHS England’s targeted lung health checks. Trial Design and Scope The Oncology Clinical Trials Office (OCTO) at Oxford will run the trial. It is designed as a dose-escalation Phase I, followed by a Phase II “precision-prevention” part. The total planned enrollment is 590 patients: 30 in Phase I and 560 in Phase II (280 per arm). The primary goals are to assess safety, determine optimal dosing, and evaluate whether the immune response can be effectively triggered. “Fewer than 10% of people with lung cancer survive their disease for 10 years or more. That must change, and that change will come from targeting lung cancer at the earliest stages,” said Professor Mariam Jamal-Hanjani, trial lead from UCL. Why This Matters Lung cancer remains the UK’s deadliest cancer by survival rate, and early detection continues to be a major challenge. By training the immune system to identify and eliminate abnormal lung cells early, before full-blown cancer develops, LungVax represents a paradigm shift in primary prevention. The vaccine does not replace existing public health measures: smoking cessation remains the most effective way to reduce lung cancer risk. But LungVax could offer a completely new layer of protection for those most vulnerable, especially as part of broader screening programmes. Backing and Future Prospects Cancer Research UK has awarded up to £2.06 million to support the four-year Phase I trial. Michelle Mitchell, Chief Executive of Cancer Research UK, emphasised the long-term vision: “By supporting the LungVax clinical trial, we will put the vaccine through the most rigorous scientific tests and take that important first step towards a world where people live longer, better lives, free from the fear of lung cancer.” The program also builds on a larger GSK-Oxford immuno-prevention partnership, which launched in early 2025, with a £50 million investment in pre-cancer vaccine research. LungVax could redefine how we approach lung cancer prevention, shifting the paradigm from reacting to disease to preventing it altogether. If the trial proves successful, this could become a powerful tool for reducing lung cancer incidence in high-risk populations. Author BioFocus Newsroom Previous Next

< Back World Health Summit 11th - 13th October, 2026 Berlin, Germany From Crisis to Resilience: Innovating for Health. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next

BAFFR-targeted CAR T cell therapy shows promise in treating relapsed follicular lymphoma. < Back PeproMene Bio Reports Complete Remission in First Follicular Lymphoma Patient from Phase 1 Trial BAFFR-targeted CAR T cell therapy shows promise in treating relapsed follicular lymphoma. PeproMene Bio, Inc . (PMB), a clinical-stage biotechnology firm focused on developing innovative treatments for cancer and immune-related conditions, announced that the first follicular lymphoma (FL) patient enrolled in its Phase 1 PMB-102 study has reached complete remission just one month after receiving therapy. The ongoing PMB-102 trial is evaluating PMB-CT01, a BAFFR-targeted CAR T cell therapy, in patients with relapsed or refractory (r/r) B-cell non-Hodgkin’s lymphoma (B-NHL). The company confirmed that this latest result brings the total number of patients achieving complete remission (CR) in the study to seven, all of whom have shown durable responses with a safety profile described as manageable. "We are excited to report that a patient with r/r FL has achieved complete remission after treatment with PMB-CT01, bringing the total to seven patients—all achieving a 100% CR rate with durable responses and a manageable safety profile. Notably, this patient had previously undergone 7 prior lines of therapy including chemoimmunotherapies, CD19 CAR T cells, an investigational trispecific antibody, and an antibody-drug conjugate (ADC). Remarkably, the patient experienced no CRS or ICANS," said Elizabeth Budde M.D., Ph.D., lead principal investigator for the study and associate professor of hematology at City of Hope, one of the country’s leading cancer research and treatment centers. Follicular lymphoma is recognized as the most prevalent slow-progressing type of B-cell non-Hodgkin lymphoma, accounting for roughly 20% of NHL cases in the United States. Although current therapies can help manage the disease, FL remains incurable, and relapse is common. With each recurrence, remission tends to become shorter, and the need for new treatment strategies becomes more urgent. "IFLI is dedicated to accelerating the development of innovative treatment options for patients with r/r FL," said Dr. Michel Azoulay, M.D., Chief Medical Officer at the Institute for Follicular Lymphoma Innovation. "I am very excited that PMB-CT01 has shown promising efficacy and safety in this first FL patient." Hazel Cheng, Ph.D., Chief Operating Officer at PeproMene Bio, added: "Most of the PMB-102 trial participants relapsed after CD19 CAR T therapy and/or presented with CD19 negative tumors. PMB-CT01 could present a viable alternative option for patients facing this challenging scenario. We are deeply committed to the development of this first-in-class BAFFR CAR T therapy and are excited to advance our study into a multi-site expansion phase that will include r/r MCL, DLBCL and FL patients." Author BioFocus Newsroom Previous Next

< Back 12th – 14th November, 2024 Boston, MA PMC Annual Personalized Medicine Conference The Annual Personalized Medicine Conference is designed to provide attendees with an opportunity to develop collaborative solutions to shared challenges in personalized medicine. Participants will exchange views with leading business executives, clinicians, journalists, and patient advocates in an intimate conference setting. Previous Register now Next

Strategic partnerships in immuno-cncology propel Innovation, highlighting ProBio’s role in the $3.3 billion LM-299 cancer therapy breakthrough. < Back GenScript’s ProBio Pioneers Exciting Innovation in Immuno-Oncology Strategic partnerships in immuno-cncology propel Innovation, highlighting ProBio’s role in the $3.3 billion LM-299 cancer therapy breakthrough. The announcement that ProBio, a subsidiary of GenScript Biotech, has successfully licensed its PD-1 new molecular entity (NME) to LaNova Medicines marks a significant milestone in the biotech industry's ongoing battle against cancer. This collaboration, bolstered by LaNova’s subsequent $3.3 billion agreement with Merck & Co. for the development of the PD-1/VEGF bispecific antibody (LM-299 program), exemplifies the transformative power of strategic partnerships and innovation in immuno-oncology. The Role of PD-1 in Cancer Therapy The licensed PD-1 molecule developed by ProBio represents a cornerstone in next-generation cancer treatments. Programmed death-1 (PD-1) inhibitors are integral in immunotherapy, empowering the immune system to recognize and combat cancer cells. By pairing PD-1 with vascular endothelial growth factor (VEGF) inhibition in a bispecific antibody, LaNova aims to create a dual-action therapy capable of modulating the tumor microenvironment while directly enhancing immune response. This approach aligns with the broader industry trend of advancing combination therapies to tackle complex cancers more effectively. ProBio’s Business Model: A Competitive Edge ProBio’s integrated Contract Development and Manufacturing Organization (CDMO) model sets it apart from competitors. By combining the development of proprietary NMEs with collaborative process development and manufacturing, ProBio positions itself as a partner of choice for biotech companies aiming to bring novel therapies to market. The success of this partnership with LaNova not only reinforces ProBio’s commitment to cutting-edge research but also showcases its ability to execute high-value deals, such as the Merck-LaNova agreement. The financial implications of this partnership are significant. Beyond immediate revenue projections, the collaboration is poised to generate long-term value by demonstrating the scalability and market potential of ProBio’s innovative molecules. This reinforces its leadership in the immuno-oncology CDMO space. Strategic Growth for GenScript and ProBio The financial boost from the LaNova-Merck deal aligns with GenScript’s strategic plans to expand ProBio’s global manufacturing footprint, particularly at its Hopewell, NJ site. Such expansions are crucial for meeting the rising demand for advanced biologics and ensuring the company remains competitive in a fast-evolving landscape. GenScript’s diversified business model, encompassing life sciences, biologics manufacturing, and synthetic biology, provides a robust platform for sustained growth. The company’s reputation, supported by its extensive customer base and substantial contributions to scientific literature, underscores its ability to deliver high-quality products and services. Implications for the Biotech Industry This development exemplifies a broader trend in the biotech industry: the increasing reliance on collaborative ecosystems to drive innovation. Companies like ProBio and LaNova leverage complementary strengths, from early-stage molecule development to late-stage commercialization, accelerating time-to-market for potentially life-saving therapies. Moreover, the agreement underscores the growing interest in immuno-oncology as a therapeutic area with both high clinical impact and lucrative market potential. The PD-1/VEGF bispecific antibody highlights the shift toward multifunctional biologics, which hold the promise of addressing unmet medical needs in oncology. The licensing of ProBio’s PD-1 molecule and the subsequent deal between LaNova Medicines and Merck exemplify the biotech industry's ability to translate scientific innovation into meaningful therapeutic advances. By fostering collaborative partnerships and strategically expanding its capabilities, GenScript’s ProBio is not only advancing the fight against cancer but also redefining the role of CDMOs in the pharmaceutical value chain. This achievement is a testament to the power of innovation and partnership in shaping the future of healthcare. Author BioFocus Newsroom Previous Next

< Back World Health Summit 11th - 13th October, 2026 Berlin, Germany From Crisis to Resilience: Innovating for Health. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next

This partnership is set to combine Asimov’s cutting-edge gene editing platform with Revopsis’s expertise in rare genetic diseases, potentially accelerating the development of next-generation gene therapies. < Back Asimov and Revopsis Therapeutics Enter Strategic Licensing Agreement to Advance Gene Therapy Solutions This partnership is set to combine Asimov’s cutting-edge gene editing platform with Revopsis’s expertise in rare genetic diseases, potentially accelerating the development of next-generation gene therapies. Innovative Collaboration to Drive Genetic Medicine Forward Asimov , renowned for its advanced work in synthetic biology and genome engineering, has partnered with Revopsis Therapeutics to enhance the development of gene therapies. Under the newly signed agreement, Revopsis will gain access to Asimov’s state-of-the-art gene-editing technologies, including its proprietary tools for precise genetic modification and optimization. Revopsis, a company focused on rare and inherited genetic disorders, is poised to leverage these advanced tools to develop innovative treatments for conditions that currently lack effective therapeutic options. As part of the agreement, Asimov will provide its technology to support the design, development, and potential commercialization of gene therapies for genetic diseases, with a particular focus on rare and hard-to-treat disorders. A Leap Forward in Genetic Medicine This strategic alliance comes at a critical time in the gene therapy landscape. With advancements in gene editing technologies such as CRISPR and other synthetic biology tools, the potential to address previously untreatable genetic conditions has never been greater. However, bringing these breakthroughs to patients requires overcoming significant technical and regulatory hurdles. The collaboration between Asimov and Revopsis seeks to address these challenges by combining their expertise to accelerate the development of safe, effective therapies. Dr. Johnathan Taylor, CEO of Asimov, expressed enthusiasm about the partnership, emphasizing how Revopsis’s mission aligns with Asimov’s vision of using synthetic biology to solve complex health problems. “Together, we aim to bring transformative treatments to patients suffering from rare genetic diseases, with the goal of improving lives and advancing the field of gene therapy,” Dr. Taylor stated. For Revopsis, the collaboration represents a significant step forward in its quest to develop gene therapies for genetic diseases with high unmet medical needs. “By incorporating Asimov’s cutting-edge gene-editing technologies, we believe we can expedite our efforts to deliver safe, effective treatments that will transform the lives of patients and their families,” said Dr. Rachel Fernandez, CEO of Revopsis Therapeutics. Impact on the Future of Gene Therapy This licensing agreement is more than just a partnership between two companies; it signals a broader trend in the biotechnology sector toward greater collaboration in the quest for solutions to rare genetic diseases. By merging expertise in synthetic biology with a deep understanding of genetic disorders, Asimov and Revopsis aim to push the boundaries of what is possible in gene therapy. While it is still early in the development process, the partnership’s potential for innovation and impact on the treatment of rare genetic diseases is considerable. The use of precise gene-editing tools to address the root causes of these disorders could transform patient outcomes and pave the way for a new era of personalized medicine. Conclusion The licensing agreement between Asimov and Revopsis Therapeutics marks an exciting milestone in the pursuit of next-generation gene therapies. By combining Asimov’s groundbreaking gene-editing technologies with Revopsis’s expertise in rare genetic diseases, this collaboration holds the promise of delivering transformative treatments that could change the lives of countless patients. As the field of gene therapy continues to evolve, this partnership is a promising step forward in the fight against genetic disorders. Author BioFocus Newsroom Previous Next

< Back World Health Summit 11th - 13th October, 2026 Berlin, Germany From Crisis to Resilience: Innovating for Health. ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next

An overview of gene therapy's history, recent breakthroughs, and what it's future may look like. < Back The Future of Medicine is in Your DNA An overview of gene therapy's history, recent breakthroughs, and what it's future may look like. On September 14th, 1990, 4-year-old Ashanthi De Silva became the first person to receive gene therapy for severe combined immunodeficiency (SCID). Her case kick- started a wave of research that spanned the 1990s, 2000s, and 2010s. Today, De Silva is living a rich, fulfilling life and is an advocate for gene therapy within the rare disease community . With a rapidly growing, multibillion dollar market , gene therapy will change the lives of thousands of people and alter the face of personalised medicine forever. The question is, how? DNA is the material that determines our physical characteristics. Defects in our DNA, called mutations, can lead to illness, disability, or death. Gene therapy is the process of modifying genetic material to treat or prevent disease, including adding, editing, or silencing genes . Way back in the 1960s, scientists had speculated that DNA could be inserted into cells to cure genetic disorders. Ashanthi De Silva had a mutant ADA gene that caused her T-cells to die off, making her prone to bacterial infections. On that fateful day in 1990, doctors introduced a healthy gene into her body, which was successful in restoring the ADA gene’s function. After De Silva, other research continued, but doctors discovered that gene therapy can trigger severe side effects, including fatal immune responses and cancer . Side effects can occur due to the biological processes underpinning gene therapy. The easiest way to insert a gene into a person’s cells is using viral vectors. Certain viruses work by entering a cell, inserting viral or disease-causing DNA into the cell’s host DNA, which produces mRNA that can code for disease-causing proteins. Gene therapy repackages this technology in the viral vector, using the technical mechanism of viruses but replacing the disease-causing viral DNA with a healthy, disease-eliminating gene . However elegant this mechanism is, it poses a risk to the body. The body can perceive the viral vector as an actual virus and may attack the carrier, which can cause a severe reaction. Since actual viruses can target multiple types of cells, the viral vector could target the wrong cell and damage perfectly healthy cells. Another major source of risk is the possibility of cancer – viral vectors could activate oncogenes, or cancer-causing genes . These risks were seen in the 1990s as gene therapies began development, and halted the progress of gene therapy until the 2010s. In the 2010s, new viral vectors were developed that were more regulated. This technology allowed the viral vectors to be specific about which cells they were targeting, when they needed to turn off or on, and tweak genes more precisely. In addition to improved viral vectors, another form of gene therapy was developed called gene editing. Gene editing allows tools such as CRISPR/CAS9 to actually cut away the defective gene and paste in a new healthy DNA sequence. A major area of recent research is the ability to use CRISPR/CAS9 to target even smaller portions of genes or individual nucleotides . Another major area of study is stem cell research, where cells that are not yet differentiated into specific cells can be edited to carry healthy genes as they grow into their final form . Today, most gene therapies fix genes outside the body and then insert the new cells into the body (ex vivo). Research is increasing on in vivo trials, where cells are edited inside the body . Gene therapy has since become a promising new form of treatment, with more financial backing from regulatory bodies and an increasing number of clinical trials dedicated to new treatments. 2025 saw some massive wins in gene therapy, including the first-ever CAR T-cell therapy for marginal zone lymphoma (a type of rare, slow-growing lymphatic cancer that presents about 7000 new cases a year). The FDA approved Breyanzi in December 2025, a drug that genetically modifies a patient’s T-cells to destroy cancer cells. Breyanzi is recommended for patients who have failed other prior treatments or have relapsed, which previously resulted in lower survival rates. The drug is life-changing: after treatment, 62.1% of patients showed no signs of lymphoma in their scans – a complete response . Another breakthrough treatment includes Waskyra, the first gene therapy for Wiskott-Aldrich Syndrome (WAS, a very rare inherited immune disorder that affects about 1 in 100,000 people, mainly boys ). Patients experience bleeding, eczema, recurrent infections, and increased immunodeficiencies. Until the FDA approved Waskyra, the only treatment for WAS was symptom management or early blood transfusions. Waskyra works by genetically correcting the patient’s blood stem cells, which are then reinfused into the patient and restore protein function. Waskyra reduced the rate of severe infections by 93% and moderate to severe bleeding events by about 60%. This life-changing treatment allows patients to participate in everyday activities with less pain and fear . That’s not all. 2025 also saw: The first cellular therapy to treat patients with severe aplastic anaemia (a rare disorder in which the bone marrow does not produce enough red blood cells ) The first gene correction for RDEB (a life-changing skin disorder ) Additional research is underway into RNA therapies and DNA therapies. Some of the most active disease areas include blood disorders like sickle-cell disease, neurological disorders such as Parkinson’s and ALS, and metabolic disorders. Clinical trials are prioritizing safety after learning from historical mistakes, and diseases and cancers that were once incurable have had therapies developed that have greatly improved the lives of patients . Though gene therapy is advancing in leaps and bounds, it is costly due to the complicated nature of the biological mechanism and the custom or mass manufacturing of viral vectors. Additionally, trials are strictly regulated inside very few clinical-grade facilities, and new therapies can take years to meet safety standards. Ethical questions are also raised, as gene therapies permanently alter a person’s biology, and the question of access and patient consent looms large . Gene therapy can offer life-changing relief for those who previously had no options available. The next decade will likely see more breakthroughs that could make gene therapy a standard form of care. Ashanthi De Silva was the first to be treated with gene therapy, but by the end of her lifetime, she will be just one of many. Author Saee Risbud , freelance contributor Previous Next

In a landmark advancement for genetic medicine, the NHS is set to offer a revolutionary gene therapy, Casgevy, to patients with transfusion-dependent beta thalassaemia. < Back NHS Introduces Groundbreaking Gene Therapy for Beta Thalassaemia In a landmark advancement for genetic medicine, the NHS is set to offer a revolutionary gene therapy, Casgevy, to patients with transfusion-dependent beta thalassaemia. The new therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics , marks a significant milestone in the treatment of this severe genetic blood disorder. Casgevy has been approved by the National Institute for Health and Care Excellence (NICE) and will be available from August 7. It is anticipated to benefit around 460 patients in England. Beta thalassaemia, a condition where the body produces insufficient haemoglobin, affects approximately 2,300 people in the UK, predominantly those of Mediterranean, Asian, or Middle Eastern descent. The disorder often leads to severe anaemia, necessitating lifelong blood transfusions and reducing life expectancy to around 50 years. Casgevy offers a potential cure by employing CRISPR gene-editing technology. The treatment involves extracting stem cells from a patient's bone marrow, modifying the genes in a lab to produce functioning haemoglobin, and reinfusing the corrected cells back into the patient. This process reprograms the cells to produce foetal haemoglobin, bypassing the genetic defect that hampers adult haemoglobin production. In clinical trials, 93% of patients did not require blood transfusions for at least a year post-treatment. "This is a historic moment for people living with beta thalassaemia," said Amanda Pritchard, NHS Chief Executive. "This therapy offers a life free from regular transfusions and the debilitating symptoms of the disorder, promising a longer and healthier life." The gene-editing tool CRISPR, which won the Nobel Prize for Chemistry in 2020, is integral to this treatment. It precisely targets and edits the DNA responsible for the haemoglobin switch from foetal to adult forms. This innovative approach not only addresses the symptoms but also targets the root cause of beta thalassaemia. The therapy's introduction follows a thorough evaluation by NICE, which considered both its costs and benefits. While the listed price of Casgevy is £1.65 million per patient, NHS England has negotiated a lower price to make it accessible. The treatment will be available at seven specialist centres across the UK, ensuring it reaches those in need efficiently. "This transformative treatment offers patients a life-changing opportunity, enabling them to repair their own cells and embrace a future free from the challenges of their condition," stated Romaine Maharaj, Executive Director of the UK Thalassaemia Society. This gene therapy not only represents a breakthrough for beta thalassaemia but also paves the way for future treatments of other genetic disorders, such as sickle cell anaemia, which is currently under negotiation for NHS approval. The rollout of Casgevy by the NHS signifies a new era in the treatment of genetic blood disorders, providing hope and improved quality of life for many patients. This advancement underscores the potential of gene therapy to address and potentially cure debilitating conditions, marking a significant achievement in medical science. Author BioFocus Newsroom Previous Next