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2025/12/04
Week 1 of December 2025 (November 27 - December 3) witnessed a landscape of innovation and challenges coexisting in the life sciences sector. Gene therapy achieved significant regulatory milestones: Novartis' SMA gene therapy received FDA approval for a new administration route, benefiting a broader patient population; CRISPR Therapeutics' cardiovascular gene editing therapy CTX310 achieved positive Phase 1 results, opening new pathways for lipid-lowering treatment. However, Intellia's ATTR amyloidosis gene editing therapy nex-z paused Phase 3 trials due to a Grade 4 liver adverse event, reminding the industry to prioritize gene editing safety. In drug approvals, FDA approved multiple oncology immunotherapies in November, while Chinese innovative drug companies accelerated internationalization: Innovent Biologics' psoriasis drug received approval, and Beida Pharma's ensartinib was accepted by the European Medicines Agency. On the policy front, the EU released a new Bioeconomy Strategy emphasizing biotechnology industrialization and market scale-up; multiple regions in China continued issuing supportive policies for cell and gene therapy; FDA vaccine regulatory policy sparked protests from former commissioners. The biotech M&A market showed significant recovery, with Q3 deal value nearly doubling from the previous quarter, reflecting renewed industry confidence.
On November 25, the U.S. FDA approved Itvisma (onasemnogene abeparvovec-brve), an intrathecally-delivered version of Novartis' spinal muscular atrophy (SMA) gene therapy, for treating SMA patients aged 2 years and older with confirmed SMN1 gene mutations. This marks the first gene replacement therapy approved for intrathecal administration and the first 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. This new administration route approval provides new treatment options for patients unable to receive intravenous Zolgensma, marking an important breakthrough in gene therapy administration technology and patient coverage. Over 3,000 patients worldwide have received this gene therapy product. citation citation
CRISPR Therapeutics' in vivo gene editing therapy CTX310 achieved significant progress in treating severe dyslipidemia. Phase 1 clinical trial results published in The New England Journal of Medicine on November 8 showed that CTX310, by targeting the ANGPTL3 gene, successfully reduced patients' triglyceride and LDL cholesterol levels. This is the first demonstration that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3 levels, leading to clinically meaningful lipid reductions. The trial enrolled patients with severe or refractory dyslipidemia, showing good safety with few adverse events. Dr. Naimish Patel, Chief Medical Officer of CRISPR Therapeutics, stated: "The publication and presentation of these Phase 1 results mark an important milestone for CRISPR Therapeutics and for the field of in vivo gene editing. For the first time, we've shown that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL." This breakthrough establishes a foundation for gene editing treatment of cardiovascular diseases and advances the company's broader cardiovascular gene editing portfolio. citation citation
In November, Intellia Therapeutics announced a temporary pause in patient dosing and screening for its Phase 3 clinical trials MAGNITUDE and MAGNITUDE-2 evaluating the gene editing therapy nexiguran ziclumeran (nex-z, also known as NTLA-2001) for transthyretin amyloidosis (ATTR). The pause followed a Grade 4 liver adverse event in a patient in the MAGNITUDE trial. The patient experienced elevated liver transaminases, occurring in less than 1% of over 650 patients enrolled in MAGNITUDE. Intellia stated the company is working closely with regulatory authorities to evaluate the event and determine a path forward for the trials. Notably, nex-z targets the TTR gene, distinct from Intellia's other gene editing therapy lonvo-z for hereditary angioedema (HAE), which targets the KLKB1 gene and uses different lipid nanoparticle delivery systems. This safety event reminds the industry that despite gene editing technology's promise, rigorous safety monitoring remains essential in clinical advancement. citation citation
According to latest market research, the global allogeneic cell therapy market is projected to reach $3.29 billion by 2032, with a CAGR of 24.5% from 2025-2032. This rapid growth is primarily driven by CRISPR/Cas9 gene editing technology breakthroughs, enabling universal cell therapy development through elimination of T-cell receptors (TCR) and HLA antigens. In November 2025, CRISPR Therapeutics initiated dosing of its first patients in the CTX112 allogeneic CAR-T program for CD19-positive malignancies, showcasing gene-edited universal cell technology. Allogene Therapeutics' ALLO-501A allogeneic CAR-T therapy achieved a 70% response rate in pivotal lymphoma trials, advancing toward biologics license application (BLA) submission. The major advantage of allogeneic cell therapy lies in "off-the-shelf" availability, eliminating patient-specific manufacturing needs and significantly reducing costs and treatment waiting times. By therapeutic area, hematological disorders occupy the largest market share, with oncology, autoimmune diseases, and neurological disorders markets also growing rapidly. citation
On November 26, Rugao Biopharma announced that its NouvNeu004 injection received FDA "Special Exemption" status and approval for an international Phase 1 clinical trial for multiple system atrophy (MSA). This follows the October 23 approval for Phase 1-3 full-cycle clinical trials by China's National Medical Products Administration, marking the world's first MSA cell therapy product achieving simultaneous US-China approval. MSA is a rare neurodegenerative disease with currently no effective treatment. NouvNeu004 brings new hope to MSA patients through cell therapy, with FDA Special Exemption status signifying accelerated pathways in clinical trial approval and regulation. This milestone achievement not only demonstrates Chinese biopharmaceutical companies' innovation strength but also brings hope to this "untreatable" disease field. citation
In November, the U.S. FDA approved Accord BioPharma's two denosumab biosimilars OSVYRTI (denosumab-desu) and JUBEREQ (denosumab-desu), serving as biosimilars to Xgeva and Prolia respectively. This marks the third pair of denosumab biosimilars to receive approval or interchangeability designation since October, following Conexxence and Bomyntra (Fresenius Kabi), and Stoboclo and Osenvelt (Celltrion USA). Other denosumab biosimilars approved earlier this year include Ospomyv and Xbryk (Samsung Bioepis), Bildyos and Bilprevda (Shanghai Henlius Biotech/Organon), and Bosaya and Aukelso (Biocon Biologics). Denosumab is a RANKL inhibitor used to treat osteoporosis and prevent skeletal-related events. The intensive biosimilar launches will significantly reduce patient treatment costs and improve drug accessibility. Additionally, on November 18, FDA approved Poherdy (pertuzumab-dpzb) injection, the first interchangeable biosimilar to Perjeta (pertuzumab), developed by Shanghai Henlius Biotech and commercialized by Organon, for treating HER2-positive breast cancer. citation citation
The FDA issued multiple oncology and supportive care approvals in November 2025, expanding treatment options for patients with breast, lung, hematologic, and solid tumor cancers. On November 19, FDA granted full approval to Amgen's Imdelltra (tarlatamab-dlle) for treating adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. This decision converted Imdelltra's prior accelerated approval to full approval based on data from the global Phase 3 DeLLphi-304 study, and the drug is now included as a Category 1 preferred treatment in NCCN guidelines. On November 20, Regeneron's Eylea HD (aflibercept 8 mg) was approved for treating macular edema following retinal vein occlusion (RVO) and for monthly dosing across approved indications. AstraZeneca's Koselugo (selumetinib) was approved for adults with neurofibromatosis type 1 (NF1) based on positive Phase 3 Komet trial results. Bayer's HYRNUO (sevabertinib) received accelerated approval for previously treated advanced HER2-mutant non-small cell lung cancer patients. Additionally, FDA approved multiple drugs for acute myeloid leukemia, including Komzifti (ziftomenib) and revumenib, providing new treatment options for refractory patients with NPM1 mutations. citation citation citation
According to PDUFA target dates, FDA is expected to make regulatory decisions on eight innovative drugs in December 2025. These include: LIB Therapeutics' Lerodalcibep, a PCSK9 inhibitor for lowering LDL cholesterol to treat atherosclerotic cardiovascular disease and primary hyperlipidemia; and multiple innovative therapies for asthma, immune diseases, and metabolic conditions. Lerodalcibep is a small binding protein (SBP) that significantly extends its half-life to 12-15 days through fusion with human serum albumin, designed to provide patients with a convenient monthly self-administered subcutaneous injection treatment. The biologics license application (BLA) is supported by the LIBerate development program including 2,900 patients across five global Phase 3 registration studies. These studies evaluated efficacy in cardiovascular disease patients or high-risk patients requiring further LDL-C reduction despite maximally tolerated statin and other oral medication therapy. December's drug approvals will bring new treatment options to patients across multiple disease areas. citation citation citation
On November 28, China's National Medical Products Administration website showed that Innovent Biologics' pimocabimab injection (IBI112, trade name: Sinmevey) received new drug approval for treating adult patients with moderate-to-severe plaque psoriasis suitable for systemic therapy. This is the first IL-23p19 targeted monoclonal antibody independently developed by a domestic enterprise, marking a new stage in China's precision psoriasis treatment. IL-23p19 is a key target in psoriasis pathogenesis, offering more durable efficacy and better safety compared to traditional IL-17 inhibitors. Sinmevey's launch fills the domestic gap in this target treatment, providing new options for moderate-to-severe psoriasis patients and demonstrating Chinese biopharmaceutical companies' innovation strength in autoimmune diseases. citation citation
On November 28, Beida Pharma announced that its controlled subsidiary Xcovery Holdings' marketing authorization application for ensartinib hydrochloride capsules (trade name: Bemeina) was accepted by the European Medicines Agency (EMA) for treating adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with ALK-positive mutations. Ensartinib is a novel, potent, highly selective next-generation ALK inhibitor. In December 2024, ensartinib received U.S. FDA approval, becoming the first small-molecule lung cancer targeted innovative drug led by a Chinese company to be approved in the United States. This EMA acceptance marks a critical step in the drug's commercialization in the European market. Beida Pharma also announced that ensartinib's marketing authorization application for post-surgical adjuvant treatment of stage IB to IIIB NSCLC patients was accepted by China's National Medical Products Administration, potentially further expanding the drug's indication scope. citation citation citation
On November 30, Biokin announced that its wholly-owned subsidiary SystImmune received a $250 million milestone payment from Bristol Myers Squibb (BMS) related to the global strategic collaboration agreement for iza-bren. Iza-bren is an EGFR×HER3 bispecific ADC drug, with its marketing authorization application for treating locally advanced or metastatic nasopharyngeal carcinoma accepted by China's National Medical Products Administration. Under the agreement, the company is eligible for up to an additional $2.5 billion in near-term contingent payments, plus up to $7.1 billion in additional payments upon achieving specific development, regulatory, and sales milestones. This significant BD progress not only brings substantial cash flow to the company but also validates its innovative drug's global competitiveness, marking Chinese innovative drug companies' rising position in international markets. citation
On November 27, the European Commission released the new "Strategic Framework for a Competitive and Sustainable EU Bioeconomy," marking the EU's bioeconomy policy shift from the 2012 strategy and 2018, 2022 reviews toward industrial deployment, market scale-up, competitiveness, and resilience. The new strategy emphasizes using biotechnology to replace fossil-based materials and products, create jobs, and lead the global shift to clean industries. The EU bioeconomy was valued at €2.7 trillion in 2023, employing over 17 million people (approximately 8% of EU jobs). Core priorities of the new strategy include: sustainable industrial biotechnology, bio-innovation-driven circular economy, R&D support, startup market integration, risk financing, regulatory simplification, and talent development. The EU aims to become a global leader in biotechnology, biomanufacturing, and sustainable bioeconomy by 2040. However, commentators note that compared to China's forward-looking strategy in its 15th Five-Year Plan emphasizing AI, bioengineering, and synthetic biology, the EU strategy remains more focused on traditional agricultural output valorization and industrial waste, with relatively conservative investment in modern engineering biology. citation citation citation
China continues advancing policy support and industrialization for cell and gene therapy. The "Proposal on Formulating the 15th Five-Year Plan for National Economic and Social Development" adopted by the Fourth Plenary Session of the 20th CPC Central Committee lists biomanufacturing as a future industry for forward-looking deployment, with cell manufacturing as important biomanufacturing content receiving high priority. During the "15th Five-Year Plan" period, Beijing's plan proposes cultivating new growth points in sixth-generation mobile communications, quantum technology, biomanufacturing, brain-computer interfaces, and advancing medical and health industrial cluster advantages. Professor Shi Ming, Deputy Director of Jiangsu Cancer Biotherapy Research Institute, notes that cell and gene technologies are hailed as "pillars of future medicine," with the State Council Development Research Center predicting the industry will enter large-scale growth in 5-10 years. Hunan Province officially implemented the "Hunan Cell and Gene Industry Promotion Regulations" in 2025, becoming the first province to legislate for cell and gene industry. Multiple regions issued intensive supportive policies, including R&D tax credits, dedicated innovation funds, and accelerated clinical trial approvals, providing clear guidance and strong guarantees for industry development. citation citation
On December 3, twelve former FDA commissioners jointly wrote a letter strongly condemning a memo sent by FDA Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad to all center staff on November 28. The memo claimed FDA scientists identified 10 child deaths linked to Pfizer and Moderna's COVID vaccines and proposed tightening vaccine approval policies. Former commissioners stated this plan would "upend core policies governing vaccine development," potentially undermining America's ability to fight infectious diseases and threatening vulnerable Americans' health. They emphasized vaccine safety monitoring should be based on scientific evidence and established procedures, not individual officials' personal opinions. This incident highlights divisions within and outside FDA regarding vaccine regulatory policy and reflects complex public health policy challenges in the post-pandemic era. The industry worries that regulatory uncertainty may impact vaccine and gene therapy R&D investment and approval timelines. citation citation
The 2025 biotechnology industry M&A activity showed significant recovery, injecting vitality into a market that experienced years of downturn. Third-quarter biotech deal value nearly doubled from the previous quarter, reflecting patent cliff pressures facing major pharmaceutical companies and ample M&A funding. Estimates suggest that by 2030, major pharmaceutical companies will face hundreds of billions of dollars in annual revenue threatened by patent expiration, with over $1 trillion in balance sheet capacity available to support M&A transactions. This environment encourages industry consolidation and rewards companies with de-risked assets. After four years of decline, many biotech companies trade at steep discounts, with industry observers still describing biotech valuations as depressed relative to long-term norms by mid-2025. This creates conditions where improving sentiment can quickly translate into performance. While biotechnology remains inherently volatile, with clinical failures, regulatory hurdles, and capital needs potentially derailing individual companies, the improving macro backdrop combined with operational discipline forced by the downturn has left many companies fundamentally stronger. FDA maintains robust approval activity, helping reduce uncertainty for companies nearing key regulatory milestones. citation
This week (November 21-27), A-share pharmaceutical index rose 2.67%, with 1.03% excess return over CSI 300; Hang Seng Biotech Index rose 4.30%, outperforming Hang Seng Tech Index by 0.52%. Sub-sector performance: APIs (+4.27%), chemical pharmaceuticals (+4.19%), pharmaceutical commerce (+2.77%), medical devices (+2.30%), biologics (+2.06%), medical services (+1.42%), traditional Chinese medicine (+1.29%). This week's strong pharmaceutical performance was driven by: Innovent Biologics' blockbuster autoimmune drug pimocabimab receiving approval; BeiGene's BCL2 inhibitor sonrotoclax receiving FDA acceptance for U.S. marketing application with priority review; small nucleic acid drug technology breakthroughs with multinational companies increasing investments. Market attention focuses on respiratory disease incidence, essential drug catalog progress, and policy environment changes such as "four-same drugs" price special governance and traditional Chinese medicine centralized procurement. Recently, Beijing and Shanghai released measures to promote high-quality medical device industry development, with expectations that national and local government supportive policies for innovative devices will continue strengthening. citation citation
A Baylor College of Medicine research team published important findings in Nature Neuroscience, discovering that boosting the protein Sox9 can help brain astrocytes clear toxic plaque buildup associated with Alzheimer's disease. In mouse models already showing memory problems, activating these cells improved cognitive performance. The treatment also reduced plaque levels over time. Sox9 protein plays a key role in astrocytes, enhancing these cells' phagocytic capacity to promote natural clearance of β-amyloid, restore blood-brain barrier function, and reverse memory loss. Additionally, Paul Scherrer Institute scientists found that spermine—a small molecule regulating many cellular processes—can guard against Alzheimer's and Parkinson's diseases. Spermine works somewhat like cheese on noodles, making certain proteins clump together and thereby rendering them harmless. These protein structures form long, thin fibers resembling spaghetti strands. Spermine's mechanism of action provides insights for developing novel neuroprotective therapies. citation citation
Lexeo Therapeutics' LX1001 is an AAV vector-based gene therapy for APOE4 homozygous Alzheimer's disease patients, delivering the APOE2 gene to the central nervous system to change patients' APOE genotype. Interim safety and efficacy data from the Phase 1/2 open-label clinical trial showed positive signals. Although current studies are not yet powered to assess efficacy, gene therapy is considered a promising direction for Alzheimer's and Parkinson's disease. Research from Toronto's Centre for Addiction and Mental Health showed experimental drug GL-II-73 has potential to restore memory and cognitive function in Alzheimer's mouse models. Meanwhile, cell therapy exploration continues, with NKGen's SNK01—a natural killer cell therapy composed of autologous NK cells that are not genetically engineered but have enhanced cytotoxicity and activating receptor expression—currently being evaluated in Phase 2 clinical trials for Alzheimer's disease efficacy. These early studies open revolutionary new pathways for neurodegenerative disease treatment. citation citation
PROTAC drugs achieved a series of R&D advances in cancer treatment in 2025. In August, the U.S. FDA accepted the new drug application for vepdegestrant, developed by Arvinas and Pfizer, for treating ER+/HER2- advanced or metastatic breast cancer patients with ESR1 mutations. Vepdegestrant is a potential "first-in-class" oral PROTAC estrogen receptor degrader that, if approved, will become the first FDA-approved PROTAC therapy, marking a new stage 20+ years after the PROTAC concept's inception. Additionally, multiple PROTAC drugs targeting prostate cancer, lung cancer, and hematologic malignancies achieved new progress. In October, Qilu Pharmaceutical presented Phase 1 clinical data for androgen receptor (AR)-targeted PROTAC drug QLH12016 at the ESMO conference. PROTAC technology utilizes the body's natural protein degradation system to selectively target and degrade disease-related proteins, promising to target previously "undruggable" targets and providing new approaches to address traditional small molecule inhibitor development challenges or overcome resistance issues. citation
Northwestern University researchers developed a novel nanostructure that supercharges CRISPR's ability to safely and efficiently enter cells, tripling gene-editing success rates. By wrapping CRISPR tools in spherical DNA-coated nanoparticles, researchers tripled gene-editing efficiency, improved precision, and dramatically reduced toxicity compared to current methods. This technological breakthrough promises to unlock CRISPR's full power to treat genetic diseases. The research findings were published in Proceedings of the National Academy of Sciences in September 2025, removing key technical barriers for gene editing therapy clinical applications. Additionally, nanotechnology achieved breakthroughs in traditional chemotherapy drug transformation, with Northwestern University researchers redesigning a common chemotherapy drug into spherical nucleic acid nanoparticle form, exponentially enhancing its potency 20,000-fold and improving safety, potentially redefining future cancer drug design and delivery. citation citation
This week's life sciences developments demonstrate a landscape balancing innovation breakthroughs with cautious advancement. Gene therapy achieved progress in expanding patient coverage and pioneering new indications, but Intellia's safety event reminds the industry of the critical importance of long-term safety monitoring. Rapid development in cell therapy and biosimilar markets provides more affordable treatment options for patients. Chinese innovative drug companies' accelerating internationalization shows the global biopharmaceutical industry landscape is undergoing profound changes. On the policy front, governments worldwide continue strengthening support for biotechnology industries, though regulatory uncertainty remains a challenge. Looking ahead, life sciences are poised to achieve more breakthroughs in precision medicine, gene editing, and cell therapy, making greater contributions to human health.
