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AUSTIN, Texas and TOKYO, Dec. 1, 2025 /PRNewswire/ -- According to DataM Intelligence, the Cystic Fibrosis (CF) Therapeutics Market Size accelerated to USD 10.60 billion in 2024, up from USD 9.22 billion in 2023, and is now projected to expand aggressively to USD 35.09 billion by 2033, recording a CAGR of 14.2% during 2025–2033. The surge is driven by transformational advancements in CFTR modulator therapies, higher treatment access across the U.S. and Europe, the expansion of personalized medicine programs, improvements in newborn screening, and stronger R&D investments targeting rare genetic diseases. Cystic fibrosis, a life-long autosomal recessive disorder caused by mutations in the CFTR gene, affects more than 162,000 patients globally, with rising diagnostic rates enabling broader therapeutic adoption. The past five years have shifted the market from symptomatic management to disease-modifying treatments, marking the most significant clinical progress in CF care in decades. Download PDF Brochure: Browse in-depth TOC on "Cystic Fibrosis Therapeutics Market" 71 – Tables 67 – Figures 192 – Pages Precision Medicine, Breakthrough Modulators & Improved Survival Rates Accelerate Market Adoption Cystic fibrosis has moved from a once-fatal childhood disease to a highly treatable chronic condition, thanks to advanced therapeutic classes. Today's market growth is being propelled by three major dynamics: 1. CFTR Modulator Innovations Are Reshaping the Standard of Care CFTR modulators such as ivacaftor, tezacaftor, elexacaftor, and combination regimens have expanded eligibility to nearly 90% of CF patients, dramatically improving lung function, BMI, respiratory exacerbation rates, and life expectancy. 2. Rising Diagnosis Rates and Earlier Intervention With neonatal screening improving globally, early treatment initiation is raising adherence and increasing long-term therapeutic demand. Countries in Europe and Asia have expanded newborn testing coverage by 12–20% YoY. 3. Growth of Symptom-Relief Therapies in Untreated CFTR Mutations A subset of patients still relies on mucolytics, bronchodilators, pancreatic enzyme replacement, and inhaled antibiotics-ensuring sustained demand across legacy CF drug segments. Market Segmentation: By Drug Class (CFTR Modulators, Pancreatic Enzyme Supplements, Mucolytics, Bronchodilators, Others) CFTR Modulators CFTR modulators represented the dominant segment in 2024, accounting for nearly 72% of total revenue (USD 7.63 billion). This class continues to see explosive uptake because of its disease-modifying capabilities and expanded label approvals across adolescents and pediatric patients. Pancreatic Enzyme Supplements Pancreatic enzyme replacement therapies (PERT) accounted for 11% (USD 1.17 billion). They remain standard-of-care for >80% of CF patients due to chronic exocrine pancreatic insufficiency. Mucolytics Mucolytics contributed 8% (USD 848 million), driven by the sustained use of inhaled agents such as dornase alfa for airway clearance. By Route of Administration (Oral, Inhaled, Parenteral) Oral Oral therapies held 69% (USD 7.31 billion) due to the dominance of oral CFTR modulators. This segment will grow fastest through 2033 driven by life-long use and expanded mutation coverage. Inhaled Inhaled therapies contributed 26% (USD 2.76 billion). This includes inhaled mucolytics, bronchodilators, antibiotics, and other respiratory-support agents still widely used in multidisciplinary CF care. By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies) Hospital Pharmacies Hospital pharmacies captured 58% (USD 6.15 billion) as CF remains primarily managed through specialty centers that coordinate multidisciplinary treatment plans. Retail Pharmacies Retail pharmacies represented 31% (USD 3.28 billion), largely driven by the growth of chronic outpatient CF management and expanded insurance coverage. Online Pharmacies Online pharmacies accounted for 11% (USD 1.17 billion) and are increasing rapidly due to home delivery convenience and improved digital prescription services for chronic diseases. Buy This Report with Year-End Offer ( Buy 1 report: Get 30% OFF | Buy 2 reports: Get 50% OFF each! Limited time offer): Regional Outlook: United States: Largest and Fastest-Expanding CF Therapeutics Market The U.S. accounted for 54% of global CF drug sales (USD 5.73 billion in 2024). U.S. Market Highlights CF patient registry size exceeds 40,000, representing 25% of global cases CFTR modulator usage penetration surpasses 88% Government and private insurers expanded coverage for next-generation modulators by 20% YoY CF care center expansions increased pulmonary inpatient capacity by 11% By 2033, the U.S. market is forecast to exceed USD 19 billion. Japan: Rapidly Modernizing CF Treatment Landscape Japan accounted for 6% (USD 636 million) of global value in 2024. Japan Market Dynamics Recent improvements in genetic screening increased CF detection rates by 16% YoY Rising access to imported CFTR modulators and specialty respiratory therapies Hospital-based CF clinics increased multidisciplinary patient management capacity by 14% Japan is on track to surpass USD 2.1 billion by 2033 as availability and early diagnosis improve. Competitive Landscape: Below are revenues and cystic-fibrosis or related respiratory/orphan-drug segment contributions based on public annual reports and investor disclosures. Vertex Pharmaceuticals Incorporated 2023 Revenue: USD 9.87 billion CF Franchise Contribution: > USD 9.3 billion (94% of total company revenue) Vertex is the global leader in CFTR modulators including Trikafta/Kaftrio, Symdeko/Symkevi, and Orkambi. F. Hoffmann-La Roche Ltd 2023 Revenue: USD 66.5 billion Respiratory & Infectious Diseases Portfolio: USD 3.4+ billion Expanding CF pipeline with inhaled and anti-inflammatory therapies. Novartis Pharmaceuticals Corporation 2023 Revenue: USD 45.4 billion Immunology & Respiratory Division: USD 8.1 billion Active in inhaled antibiotic programs and airway clearance technologies. Teva Pharmaceuticals USA, Inc. 2023 Revenue: USD 15.8 billion Respiratory Portfolio Contribution: USD 1.7 billion Strong presence in inhalation therapies supporting CF symptom relief. Gilead Sciences, Inc. 2023 Revenue: USD 27.1 billion Antiviral & Anti-Infective Division: USD 5.8 billion Previously developed Cayston (inhaled aztreonam), widely used for chronic lung infections in CF patients. Buy This Report with Year-End Offer ( Buy 1 report: Get 30% OFF | Buy 2 reports: Get 50% OFF each! Limited time offer): The Decade Ahead: What will Define the Future of Cystic Fibrosis Therapeutics The Cystic Fibrosis market is on track for the fastest evolution in its history, driven by: Next-gen CFTR modulators targeting rare mutations Combination RNA therapies for non-responders Gene-editing and gene-replacement programs, particularly using CRISPR Inhaled biologics addressing persistent lung infection Improved lipid nanoparticle (LNP) delivery systems enabling organ-specific targeting Pediatric expansion increasing long-term treatment demand Real-world evidence integration improving precision dosing By 2033, cystic fibrosis care will be defined by personalized molecular therapies, multi-modality regimens, and potentially curative genetic treatments-setting the stage for the CF therapeutics market to become one of the most valuable rare-disease drug markets globally. Related Report: Duchenne Muscular Dystrophy Therapeutics Market Size to Surge from US$2.19 B in 2024 to US$6.64 B by 2033 - 13.2% CAGR. Marfan Syndrome Treatment Market Size to Grow from US$203.5 M in 2023 to US$312.6 M by 2031 - Driven by Rising Diagnosis & Advanced Therapeutic & Surgical Care. Huntington's Disease Market Size to Grow from US$456 M in 2024 to US$3.23 B by 2033 - >7.5% CAGR Signals Surge in Therapeutic & R&D Activity. Sickle Cell Anaemia Testing Market Share to Grow at 12% CAGR - From US$0.43 B in 2024 to Over US$1.5 B by 2032. About DataM Intelligence DataM Intelligence is a renowned provider of market research, delivering deep insights through pricing analysis, market share breakdowns, and competitive intelligence. The company specializes in strategic reports that guide businesses in high-growth sectors such as nutraceuticals and AI-driven health innovations. To find out more, visit or follow us on Twitter, LinkedIn, and Facebook. Contact: Sai Kiran DataM Intelligence 4market Research LLP Ground floor, DSL Abacus IT Park, Industrial Development Area Uppal, Hyderabad, Telangana 500039 USA: +1 877-441-4866 Email: [email protected] Logo: SOURCE DataM Intelligence 4 Market Research LLP 21% more press release views with Request a Demo

摘要：疫苗是人类对抗传染病的终极武器，但传统研发模式往往耗时数年、耗资数十亿，还面临成功率低的困境。如今，人工智能（AI）的介入正在彻底改变这一现状。从精准筛选抗原、预测病毒关键靶点，到设计更安全的佐剂、优化临床试验，AI 技术贯穿疫苗研发全流程，不仅将研发周期从数年缩短至数月，还能打造个性化、广谱性疫苗。本文将用通俗语言拆解 AI 如何成为疫苗研发的 “超级大脑”，同时聊聊这项技术面临的挑战与未来可能。 一、传统疫苗研发：一场 “耗时耗力” 的持久战 提起疫苗，我们都知道它能保护我们远离天花、脊髓灰质炎等致命疾病。但很少有人了解，一款疫苗从实验室走到我们的手臂，往往要经历一场漫长的 “闯关游戏”。 传统疫苗研发通常要经过病原体分离、抗原筛选、免疫原 formulation、临床前测试和临床试验等多个阶段，整个过程少则 5 年，多则十几年。比如 HPV 疫苗从最初研究到获批上市，足足花了 15 年；而研发一款结核病疫苗，至今仍因病原体复杂、抗原难以确定而进展缓慢。 更让人头疼的是，传统研发充满了 “不确定性”。抗原筛选靠反复试验，临床试 - 验成功率不足 20%，加上生产工艺复杂、 regulatory 审批严格，一款疫苗的研发成本动辄数十亿美金。在疫情爆发时，这种慢节奏更是致命 —— 当传统疫苗还在实验室摸索时，病毒可能已经全球蔓延。 表1 传统疫苗研发的主要挑战及实例 二、AI 登场：疫苗研发的 “超级加速器” 就在传统研发模式举步维艰时，AI 技术带来了突破性改变。简单说，AI 就像给疫苗研发团队配备了一个 “超级大脑”，能处理海量数据、精准预测结果，让整个过程又快又准。 AI 在疫苗研发中的核心作用，体现在四个关键环节：1. 抗原筛选：从 “大海捞针” 到 “精准定位” 抗原是疫苗的核心，能刺激人体产生免疫反应。传统方法筛选抗原就像 “大海捞针”，而 AI 能快速分析病毒基因组数据、蛋白质结构，在短时间内锁定最具免疫活性的抗原。 比如在新冠疫情期间，AI 算法通过分析 SARS-CoV-2 病毒的基因组和蛋白质结构，迅速锁定了刺突蛋白作为核心抗原，为 mRNA 疫苗的快速研发奠定了基础。对于疟疾这种复杂疾病，AI 通过机器学习算法分析大量病原体数据，成功筛选出关键抗原，让疫苗研发周期缩短了数年。2. 表位预测：锁定病毒 “致命弱点” 表位是抗原上真正能被免疫系统识别的关键片段，相当于病毒的 “致命弱点”。AI 能通过深度学习、隐藏马尔可夫模型等技术，精准预测出病毒的 B 细胞表位和 T 细胞表位，让疫苗能针对性激发免疫反应。 这种精准预测带来了两大突破：一是能研发出 “广谱疫苗”，比如针对流感病毒的 AI 预测模型，能识别不同毒株共有的保守表位，有望研发出无需每年更新的通用流感疫苗；二是能打造 “个性化疫苗”，通过分析患者的基因数据，设计出贴合个人免疫特征的疫苗，尤其在癌症免疫治疗中潜力巨大。3. 佐剂设计：给疫苗 “加 buff” 佐剂是疫苗的 “好帮手”，能增强免疫反应、延长保护时间。但传统佐剂筛选效率低，还可能存在安全风险。AI 通过虚拟筛选、分子对接等技术，从海量化合物库中快速筛选出安全有效的佐剂候选物。 AI 还能通过结构 - 活性关系模型分析佐剂分子的结构与功能，设计出更安全、更有效的佐剂。比如针对新冠疫苗的佐剂优化，AI 通过模拟佐剂与免疫受体的相互作用，提升了疫苗的免疫原性和稳定性。4. 临床试验优化：让试验更高效 临床试验是疫苗研发的 “最后一道关卡”，传统试验存在招募难、周期长、成本高的问题。AI 能通过分析电子健康记录和基因组数据，快速找到符合条件的受试者，提高招募效率；还能模拟不同试验方案的效果，优化试验设计，减少不必要的资源浪费。 需要注意的是，AI 并不是要取代传统临床试验，而是作为 “辅助工具” 提升效率。只有通过严谨的临床验证，才能确保疫苗的安全性和有效性。 图1 AI 在疫苗研发中的核心应用环节 表2 疫苗研发中常用的 AI 工具及功能 三、AI 疫苗的未来：精准、快速、个性化 随着 AI 技术的不断发展，疫苗研发正在朝着三个方向进化：1. 研发速度 “质的飞跃” 传统疫苗研发需要数年时间，而 AI 能将这一周期缩短至数月甚至数周。在新冠疫情中，AI 驱动的 mRNA 疫苗从研发到获批仅用了不到一年时间，创造了疫苗研发的纪录。未来，面对突发传染病，我们可能在病毒测序完成后不久，就能快速研发出针对性疫苗。2. 个性化疫苗成为可能 每个人的基因和免疫状况不同，对疫苗的反应也存在差异。AI 能通过分析个人的基因组、转录组等数据，设计出贴合个人免疫特征的疫苗。这种个性化疫苗不仅能提高免疫效果，还能减少不良反应，尤其适合老年人、免疫功能低下等特殊人群。3. 广谱疫苗有望攻克 “变异病毒” 流感、HIV 等病毒容易发生变异，导致传统疫苗效果受限。AI 能通过分析病毒的进化规律和保守序列，设计出能覆盖多种毒株的广谱疫苗。比如通用流感疫苗、通用冠状病毒疫苗等，一旦成功研发，将彻底改变我们应对病毒变异的方式。四、机遇背后：AI 疫苗研发面临的挑战 尽管 AI 在疫苗研发中表现亮眼，但要实现大规模应用，还需要克服几个关键挑战： 首先是数据问题。AI 模型的性能依赖大量高质量数据，但目前疫苗研发相关数据往往分散、不完整，甚至存在偏差。只有建立标准化的数据共享平台，才能让 AI 模型发挥最大作用。 其次是模型可解释性。很多 AI 模型被称为 “黑箱”，难以解释预测结果的科学依据。在疫苗研发这种关乎公共健康的领域，模型的可解释性至关重要，需要科研人员不断优化算法，让 AI 的决策过程更透明。 最后是监管与伦理。现有疫苗监管框架是为传统研发模式设计的，AI 驱动的疫苗研发需要更灵活的监管政策。同时，还要确保 AI 技术的公平应用，避免因资源分配不均导致疫苗可及性差距扩大。 图2 AI 疫苗研发的挑战与解决方案五、结语：AI 让疫苗研发更高效、更公平 从爱德华・詹纳发明牛痘疫苗至今，疫苗已经拯救了数十亿人的生命。而 AI 技术的加入，正在给疫苗研发带来一场前所未有的革命 —— 它让研发更快速、更精准、更具针对性，也让我们在面对传染病威胁时更有底气。 当然，AI 并不是万能的，它需要与生物技术、免疫学等学科深度融合，还需要科研人员、企业和监管机构的密切合作。但不可否认的是，AI 已经成为疫苗研发的 “核心驱动力”，正在改写全球公共卫生的格局。 未来，我们或许不用再为突发疫情恐慌，不用再担心病毒变异导致疫苗失效。随着 AI 技术的不断进步，更安全、更有效、更公平的疫苗将惠及每一个人，让人类在与传染病的斗争中占据更有利的位置。 识别微信二维码，添加生物制品圈小编，符合条件者即可加入 生物制品微信群！ 请注明：姓名+研究方向！ 版 权 声 明 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。