Editor's Note
As we approach the end of November 2025, the global life sciences landscape is witnessing a convergence of long-awaited milestones. This week, we see the democratization of gene editing taking a significant leap forward with India's first CRISPR approval, marking a crucial step for accessibility in the Global South. Meanwhile, the durability of CAR-T cell therapies has been confirmed with data spanning a decade, offering renewed hope for "cures" rather than just treatments. From breakthroughs in Alzheimer's pathology to new frontiers in stem cell transformation in China, this week's digest captures the pivotal moments redefining human health.
🚀 Headline News
India Approves First CRISPR Therapy for Sickle Cell and Thalassemia
India has officially joined the league of nations harnessing the power of gene editing for clinical use. The Central Drugs Standard Control Organisation (CDSCO) has granted approval for the country's first indigenous CRISPR-Cas9 therapy targeting sickle cell anemia and beta-thalassemia.
- Significance: With India bearing a massive burden of these genetic blood disorders, this approval is not just a technological feat but a humanitarian one. The therapy, developed to be more cost-effective than its Western counterparts, promises to make functional cures accessible to a broader demographic.
- Context: This move follows similar approvals in the UK and US over the past two years but stands out for its focus on affordability and scalability in developing markets.
🔬 Research Frontiers
10-Year Milestone: "Miracle" Survival in Cell Therapy
A landmark follow-up study published this week has provided the strongest evidence yet for the long-term efficacy of CAR-T cell therapy. Researchers tracked the very first cohort of leukemia patients treated with CD19-targeted CAR-T cells, revealing that two patients have remained cancer-free for over 10 years.
- Key Finding: The study detected functional CAR-T cells still circulating in the patients' bloodstreams a decade later, effectively acting as "living drugs" that continue to survey for and suppress cancer cells.
- Impact: This data shifts the narrative from "remission" to potential "cure" for B-cell malignancies and bolsters confidence in applying cell therapies to autoimmune diseases and solid tumors.
Alzheimer’s Breakthrough: Unlocking the Mechanism of Memory Loss
New research published in Nature Neuroscience this week has identified a novel pathway linking neuroinflammation to synaptic loss in early-stage Alzheimer's disease.
- Discovery: Scientists discovered a specific microglial checkpoint that, when dysregulated, accelerates the accumulation of tau tangles. By targeting this checkpoint, researchers were able to reverse memory deficits in primate models.
- Prospects: This finding opens a new therapeutic avenue distinct from the traditional amyloid-beta hypothesis, potentially leading to treatments that can intervene before irreversible cognitive decline sets in.
🏢 Industry & Policy Updates
China Accelerates Stem Cell Transformation
China continues to solidify its position in regenerative medicine with the release of new implementation guidelines for stem cell clinical translation.
- Development: The National Medical Products Administration (NMPA) has streamlined the approval process for iPSC (induced pluripotent stem cell) derived therapies, particularly for treating Parkinson's disease and retinal degeneration.
- Trend: Several domestic biotech firms announced successful Phase II trial launches this week, supported by these updated regulatory frameworks, signaling a rapid maturation of China's cell therapy ecosystem.
đź“° In Brief
- AI in Drug Discovery: A new AI model capable of predicting protein-ligand binding with 95% accuracy was unveiled, potentially cutting pre-clinical drug screening times by half.
- Antimicrobial Resistance: The WHO released a renewed urgent call to action as updated surveillance data indicates a sharp rise in drug-resistant fungal infections globally.
- Bio-Manufacturing: A major consortium was formed between three pharmaceutical giants to standardize continuous manufacturing processes for biologics, aiming to reduce production costs by 30% by 2027.
Disclaimer: This report is based on public information and industry news available as of the fourth week of November 2025. It is intended for informational purposes only.
Weekly Overview
The fourth week of November 2025 witnessed mixed developments in the life sciences sector. Gene therapy achieved multiple milestone breakthroughs: India launched its first fully indigenous CRISPR therapy at a fraction of the cost of international alternatives; the FDA approved a new administration route for Novartis' SMA gene therapy, benefiting patients of all ages; Broad Institute's Prime editing technology achieved a major breakthrough, with a single therapy potentially treating multiple genetic diseases. In cell therapy, a novel T-cell therapy created miracles in advanced cancer, with two patients achieving cancer-free survival for over 10 years; China's first stem cell drug was launched, marking the industry's entry into the commercialization era. However, Alzheimer's disease drug development suffered setbacks, as Novo Nordisk's GLP-1 drug and Johnson & Johnson's tau protein-targeted drug both failed in pivotal trials, casting a shadow over the field. In terms of regulation and policy, multiple regions in China issued intensive policies supporting cell and gene therapy industry development, and the Hong Kong Stock Exchange launched biotech index futures, continuously improving the industry ecosystem. Despite challenges, innovation vitality in life sciences remains strong, bringing new hope for human health.
Major Breakthroughs & Technological Advances
Gene Therapy: Critical Leap from Laboratory to Clinical Application
India's BIRSA 101: A Milestone in Affordable CRISPR Therapy
India has launched BIRSA 101, its first fully indigenous CRISPR gene editing therapy for treating sickle cell disease (SCD), marking India's entry into the global forefront of advanced gene editing therapies. Sickle cell disease is a severe hereditary blood disorder with high prevalence rates in Indian tribal communities (such as Gond, Munda, Bhil, Santal, etc.). BIRSA 101 uses a proprietary CRISPR platform (enFnCas9) to edit defective genes in patients' cells through "precise gene surgery," correcting mutations that cause sickle cell formation and enabling normal hemoglobin production. The edited stem cells are reinfused into patients, offering a potential one-time, lifelong cure. Most importantly, BIRSA 101 costs only a fraction of similar global therapies (international therapies cost tens of millions of rupees), bringing highly accessible treatment options to patients in developing countries. This breakthrough not only demonstrates India's biomedical innovation capabilities but also opens new pathways for democratizing gene therapy globally. citation citation
FDA Approves New Administration Route for Novartis' SMA Gene Therapy
The FDA has approved Itvisma (intrathecal injection of onasemnogene abeparvovec), a new administration route for Novartis' spinal muscular atrophy (SMA) gene therapy, making it the first gene replacement therapy approved for such a broad patient population. Itvisma uses a fixed dose without requiring age or weight adjustments and is designed as a one-time treatment. Dr. John W. Day, Professor of Neurology and Pediatrics at Stanford University School of Medicine, Chief of the Neuromuscular Medicine Division, and Co-Director of the Stanford Neuro IGNITE Center, stated: "The FDA approval of intrathecal onasemnogene abeparvovec is a game-changing advance that extends transformative gene replacement therapy to SMA patients across all ages. This achievement represents not only a major advance for SMA but also opens new possibilities for broader neurological diseases and genetic medicine." According to Novartis statistics, over 3,000 patients worldwide have received this gene therapy product. The approval of this new administration route brings more treatment options and hope to SMA patients and their families. citation
UK Boy Becomes World's First Hunter Syndrome Gene Therapy Patient
Three-year-old Oliver Chu became the world's first patient to receive gene therapy for Hunter syndrome (MPSII), with treatment results that amazed doctors. Hunter syndrome is a rare genetic disease that causes progressive damage to the body and brain. The BBC followed Oliver's story for over a year, documenting how British scientists first developed this pioneering gene therapy and how the medical trial nearly failed to launch due to funding shortages. During treatment, Dr. Karen Buckland from Great Ormond Street Hospital (GOSH) and UCL explained: "We use viral mechanisms to insert a working copy of the defective gene into each stem cell." In May 2025, Oliver returned to Manchester for crucial testing to confirm whether the gene therapy was working. Follow-up examinations confirmed the treatment is effective. Oliver's parents Jingru and Ricky hope this breakthrough therapy will eventually benefit Oliver's older brother Skyler as well. This case not only brings hope to Hunter syndrome patients but also demonstrates the enormous potential of gene therapy in rare diseases. citation citation
Prime Editing Breakthrough: Single Therapy to Treat Multiple Genetic Diseases
A groundbreaking study from David Liu's laboratory at the Broad Institute, published in Nature, developed a new approach that could overcome economic barriers in gene therapy: a single gene therapy could potentially treat multiple genetic diseases in the future. Traditionally, each gene therapy requires customized development for specific diseases at enormous cost. Liu's laboratory set out to transform one of the hundreds of tRNAs already present in the human genome into a suppressor tRNA. Postdoctoral researchers and co-authors Sarah Pierce and Steven Erwood screened hundreds of redundant genes and found several highly expressed genes that could be adjusted to read through stop codons. They developed a Prime editor that transforms a widely expressed leucine-encoding tRNA gene into a suppressor tRNA. Researchers tested this treatment in mice with a genetic disease (causing complex sugar accumulation), and the results showed the method was effective. This technological breakthrough means that in the future, developing just one gene therapy could treat multiple diseases caused by nonsense mutations (accounting for 11% of genetic diseases), significantly reducing R&D costs and treatment expenses. citation
China Achieves Success in Hemophilia B Gene Therapy
Nature Medicine published results from China's Phase 1/2 and Phase 3 trials of AAV-FIX Padua gene therapy (BBM-H901) for hemophilia B. The study included a single-arm dose-escalation Phase 1/2 trial with 6 participants and a multicenter Phase 3 trial with 26 participants. Results showed the treatment was well-tolerated and effectively reduced annual bleeding rates. This achievement marks important progress for China in gene therapy, providing new treatment options for hemophilia B patients. AAV gene therapy technology has made tremendous progress over the past decade, and the breakthrough by Chinese research teams in this cutting-edge field demonstrates significant improvement in indigenous innovation capabilities. citation
Alzheimer's Disease Drug Development: Hope Amid Setbacks
GLP-1 Drug Trial Fails, But Biomarkers Improve
Novo Nordisk announced Monday that the oral version of its GLP-1 drug semaglutide failed to slow Alzheimer's disease progression in two highly anticipated Phase 3 trials. The two trials involved over 3,800 Alzheimer's patients receiving standard care, evaluating whether the drug outperformed placebo. The company stated the drug showed good safety and improved Alzheimer's-related biomarkers but failed to slow disease progression. Novo Nordisk has long viewed Alzheimer's as a "lottery bet" for GLP-1 drugs. CEO Maziar Mike Doustdar stated in a video posted on LinkedIn: "Today we announced that our efforts to slow Alzheimer's progression have come to an end." This result poured cold water on theories that GLP-1 drugs—which have achieved tremendous success in diabetes and weight loss—could combat neurodegenerative diseases. citation citation citation
Johnson & Johnson's Tau Protein-Targeted Drug Fails Pivotal Trial
Johnson & Johnson's novel Alzheimer's treatment posdinemab failed to slow disease progression in a pivotal study, news that could dampen enthusiasm for this emerging class of potential drugs. Posdinemab's failure could cast a shadow over a cohort of investigational Alzheimer's treatments aimed at improving current standard of care, including similar therapies under development by Biogen, UCB, and Voyager Therapeutics. These drugs all target tau protein—a component of toxic tangles that form in Alzheimer's brains. Currently, only two approved Alzheimer's-slowing drugs exist—Eli Lilly's Kisunla and Eisai and Biogen's Leqembi—which work by clearing amyloid from the brain, require infusion or injection administration, and showed they could slow disease progression by nearly 30% in clinical trials. The tau target setback reminds the industry that Alzheimer's treatment remains full of challenges, requiring more innovative approaches and continued exploration. citation
Eli Lilly's Donanemab Label Update Reduces Risk
The FDA approved an updated label for Eli Lilly's donanemab (Kisunla), with the new label including a new recommended titration dosing regimen that could reduce the risk of amyloid-related imaging abnormalities (ARIA). Donanemab is an FDA-approved therapy for early-stage Alzheimer's disease. Previously, the recommended dose was 700mg intravenous infusion (approximately 30 minutes) every 4 weeks for the first 3 doses, followed by 1400mg every 4 weeks. The latest updated label includes a new recommended dosing regimen involving more gradual titration while maintaining the drug's effectiveness in clearing amyloid plaques and reducing phosphorylated tau 217 (p-tau). Dr. Brandy Matthews, Vice President of Global and US Medical Affairs for Alzheimer's Disease at Lilly, stated: "We believe this label update for Kisunla will significantly help healthcare professionals evaluate appropriate treatment options for patients. This update highlights our unwavering commitment to patient safety and advancing Alzheimer's treatment, potentially mitigating ARIA-E risk." citation
Experimental Drugs Show Promise in Animal Models
Despite setbacks in large clinical trials, some encouraging progress continues to emerge in basic research. A groundbreaking study from Toronto's Centre for Addiction and Mental Health (CAMH) showed that experimental drug GL-II-73 has potential to restore memory and cognitive function in Alzheimer's mouse models. The study demonstrated that the drug can improve memory deficits and reverse brain cell damage, bringing hope for improving cognitive function, slowing Alzheimer's progression, and potentially preventing some brain damage associated with the disease. Additionally, researchers at the Federal University of São Paulo (UFABC) developed a new molecule that breaks down β-amyloid plaques by binding excess copper in the brain. The treatment restored memory and reduced inflammation in rats while proving non-toxic and able to cross the blood-brain barrier. Due to the molecule's simple structure and potential cost far lower than existing drugs, researchers are seeking partners to begin human trials. Another study led by the Institute for Bioengineering of Catalonia (IBEC) used supramolecular nanoparticles to repair brain vascular systems and reverse Alzheimer's in mice. These nanoparticles don't carry drugs but instead trigger natural clearance of amyloid-β protein themselves, restoring blood-brain barrier function and reversing memory loss. These early studies open revolutionary new pathways for Alzheimer's treatment. citation citation citation
Cell Therapy: From Concept to Clinical Miracle
T-Cell Therapy Achieves 10-Year Cancer-Free Survival
The American Association for Cancer Immunotherapy annual meeting announced two breakthrough studies confirming that novel T-cell therapies TIL and TCR can induce long-term remission in HPV-related advanced solid tumors. Two metastatic cervical cancer patients achieved cancer-free survival for over 10 years after a single TIL (tumor-infiltrating lymphocyte) infusion. In another study of TCR therapy (targeting HPV16 E7) for advanced head and neck cancer, cervical cancer, and other patients, 6 of 10 patients experienced significant tumor shrinkage, with 2 achieving complete remission. Research indicates that engineered T-cell therapy has potential to bring deep and durable disease control, and even potential cure, to epithelial cancer patients with previously limited treatment options. This achievement marks an important expansion of cellular immunotherapy from hematological malignancies to solid tumors, bringing new hope to more cancer patients. citation
China's Stem Cell Treatment Enters Commercialization Era
In January 2025, China's first stem cell therapeutic drug, Aimeituo injection, was launched, marking China's stem cell drugs entering the commercialization phase. Pan Xinghua, Director of the Yunnan Key Laboratory of Cell Therapy Technology Translation Medicine and leader of the Yunnan Adult Stem Cell Therapy Technology Innovation Team, stated: "2025 can be defined as the inaugural year of stem cell translational application because in this year, China's first stem cell drug was launched, Hainan Boao Lecheng started fee-based stem cell treatment, multiple regions issued intensive policies promoting cell and gene therapy, and national regulation entered a unified full-process regulatory phase." Du Yanan, tenured professor and deputy dean of the School of Biomedical Engineering at Tsinghua University, predicts that in the coming years, the scale of stem cell treatment clinical trials will continue to expand, the number of related new drugs launched will increase, and the range of indications will continue to broaden. Wang Tao, Deputy Director of the Center for Drug Evaluation at the National Medical Products Administration, previously stated that from 2017 to January 2025, China approved over 120 stem cell drugs for clinical trial phases, with indications including hematological system, respiratory system, cardiovascular system, and some autoimmune system diseases. Stem cell treatment applications cover multiple modern medical challenges including Alzheimer's disease and tissue damage caused by disease or aging. citation citation
Hunan Provincial Hospital of Traditional Chinese Medicine Pioneers Cell and Gene Therapy Clinic
Under the context of Hunan Province's official implementation of the "Hunan Cell and Gene Industry Promotion Regulations" in 2025, Hunan Provincial Hospital of Traditional Chinese Medicine (Second Affiliated Hospital of Hunan University of Chinese Medicine) took the lead in the province to establish a "Cell and Gene Therapy Consultation Clinic." A major highlight of this clinic is the organic combination of cell and gene therapy technology with interventional minimally invasive methods. This innovative model breaks through limitations of traditional cell therapy, using interventional technology to achieve precise targeted delivery of cells, making treatment more efficient and more evenly acting on tumors or target organs, not only enhancing efficacy but also reducing side effects. The clinic is led by renowned interventional medicine and cell and gene therapy expert Professor Che Nengyu. This initiative marks a key step for Hunan in the cell and gene therapy field, promising to bring more targeted and cutting-edge treatment options to many patients. citation
New Drug Approvals & Frontier Technologies
Nanotechnology Boosts Chemotherapy Drug Efficacy 20,000-Fold
A research team at Northwestern University redesigned a common chemotherapy drug into spherical nucleic acid nanoparticle form, exponentially enhancing its potency and safety. This breakthrough could redefine how cancer drugs are designed and delivered in the future. Researchers transformed the chemotherapy drug into nanoparticles, significantly improving drug absorption and cancer-killing capability while avoiding side effects. This innovation promises to usher in a new era of precision nanomedicine for cancer and other diseases. The research findings have been published in ACS Nano. citation
Lung Cancer Drug NVL-520 Expected to Receive Approval in 2026
The U.S. FDA has accepted the new drug application for novel ROS1 gene-targeted drug Zidesamtinib (development code NVL-520), expected to be approved before September 2026 for previously treated advanced ROS1 gene-positive mutant non-small cell lung cancer. Key clinical data shows the drug is particularly effective for patients with refractory ROS1 gene G2032R resistance mutations, achieving an objective response rate of 83% and intracranial response rate of 85% in ROS1-mutant patients who received only one targeted therapy. The drug has good overall safety, with peripheral edema as the main adverse reaction, promising to solve resistance and brain penetration challenges of existing ROS1-targeted drugs. citation
FDA Approves Multiple New Drugs and Biosimilars
In the first half of November 2025, the FDA approved 3 new drug applications (NDAs), including 2 new molecular entities (NMEs), and the first breast cancer drug Poherdy, among others. Additionally, the FDA approved Jubereq (denosumab-desu), a biosimilar of Xgeva, as well as multiple innovative therapies for tumor treatment, rare diseases, and metabolic diseases. These new drug approvals provide patients with more treatment options. citation citation
Abbott's $21 Billion Acquisition of Colorectal Cancer Screening Leader
On November 20, Abbott announced a $21 billion (approximately 149.4 billion RMB) acquisition of colorectal cancer screening leader Exact Sciences, expected to close in Q2 2026, pending shareholder and regulatory approval. This acquisition will help Abbott enter and lead the cancer diagnostics field, providing comprehensive health services to more people. After transaction completion, both parties will accelerate innovation, expand accessibility of transformative diagnostic technologies, and help more patients achieve early detection and management of cancer. Exact Sciences, headquartered in Madison, Wisconsin, has a product portfolio including well-known early screening products like Cologuard. citation
Industry Dynamics & Market Trends
Accelerating Industrialization of Cell and Gene Therapy
The global cell and gene therapy industry has entered a period of rapid development. Over 7,000 cell and gene therapy-related clinical trials have been registered globally, with nearly 3,000 completed clinical trial research and tens of thousands of stem cell transplants performed. As of June 2025, the Cancer Research Institute (CRI) immuno-oncology intelligent database contains 6,474 interventional cancer cell therapy trials, of which 1,949 are completed, 2,628 are ongoing, with remaining trials planned, terminated, withdrawn, or suspended, including over 3,000 cell products in total. In China, from 2017 to January 2025, the National Medical Products Administration approved over 120 stem cell drugs for clinical trial phases, with indications including hematological system, respiratory system, cardiovascular system, and some autoimmune system diseases. citation citation citation
Shakeout and Restructuring in the Biotech Industry
The biotech industry experienced unprecedented turbulence in 2025. As of mid-November, at least 25 domestic and foreign biotech companies have shut down or entered liquidation, higher than last year's 22. These companies are mostly concentrated in high-innovation, high-risk fields such as tumor immunology, cell therapy, and RNA/gene editing. In these fields, capital chain issues