[Latest] Gene Therapy Market Size US$ 35.91 Billion by 2033: New Technologies and Strategic Japan Developments

Gene Therapy Market size reached US$ 4.80 Billion in 2024 and is expected to reach US$ 35.91 Billion by 2033, growing at a CAGR of 23.1% during the forecast period 2025-2033.

➠ Gene Therapy Updates - Japan

➠ Chugai receives approval for Duchenne Muscular Dystrophy (DMD) gene therapy
✦ In May 2025, Chugai Pharmaceutical secured conditional approval from Japan's Ministry of Health, Labour and Welfare for ELEVIDYS (delandistrogene moxeparvovec), a one-time intravenous gene therapy targeting ambulatory boys aged 3-7 with Duchenne muscular dystrophy. Eligibility includes specific genetic profiles and antibody status

➠ Optogenetic gene therapy enters clinical trial for retinitis pigmentosa
✦ In February 2025, Keio University and partners began a clinical trial using optogenetic gene therapy to restore light sensitivity in patients with genetic retinitis pigmentosa. The trial's first patient was treated on February 6, with plans to enroll up to 15 individuals

➠ CUL5 gene suppression enhances CAR‐T cancer therapies
✦ Researchers at Nagoya University discovered that reducing CUL5 gene activity can improve CAR‐T cell persistence and antitumor effect. In mouse models of B‐cell lymphoma, this modification led to better tumor shrinkage and reduced relapse risk .

➠ Japan set to review first Hemophilia B gene therapy
✦ In February 2025, an advisory panel met to evaluate Pfizer's Beqvez (fidanacogene elaparvovec), potentially marking Japan's first approved gene therapy for Hemophilia B

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Gene Therapy Market Overview

Gene therapy is a medical approach that involves altering the genetic material of a person's cells to treat or prevent diseases by correcting or replacing faulty genes.

Research Methodology:

The global Gene Therapy Market research report is based on a combination of primary and secondary data sources. The study carefully analyzes various factors influencing the industry, including government regulations, market dynamics, competitive landscape, historical trends, current conditions, and technological progress. It also evaluates future developments, related industry trends, market volatility, growth prospects, potential obstacles, and emerging challenges.

Segment Covered in the Gene Therapy Market:

By Approach: In-Vivo, Ex-vivo

By Vector Type: Viral Vectors, Non-Viral Vectors

By Technique: Gene Addition, Gene Silencing, Gene Editing

By Application: Rare Diseases, Musculoskeletal Conditions, Blood Disorders, Oncology, Ophthalmology, Others

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List of the Key Players in the Gene Therapy Market:

Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics group., Krystal Biotech, Inc., and among others.

Industry Development:

Likewise, in March 2024, Orchard Therapeutics, now a part of Kyowa Kirin, announced its U.S. launch plans for Lenmeldy (atidarsagene autotemcel)-the first FDA-approved gene therapy for treating children with early-onset metachromatic leukodystrophy (MLD). MLD is a rare and aggressively progressive neurometabolic disorder, affecting approximately 1 in 100,000 live births, and is ultimately fatal without intervention.

Regional Analysis for Gene Therapy Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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The Report Includes:

➡ A descriptive analysis of demand-supply gap, market size estimation, SWOT analysis, PESTEL Analysis and forecast in the global market.

➡ Top-down and bottom-up approach for regional analysis

➡ Go-to-market Strategy.

➡ Neutral perspective on the market performance.

➡ Customized regional/country reports as per request and country level analysis.

➡ Potential & niche segments and regions exhibiting promising growth covered.

People Also Ask:

➠ What are the global sales, production, consumption, imports, and exports in the Gene Therapy market?

➠ Who are the top manufacturers, and what are their capacity, production, sales, pricing, and revenue stats?

➠ What key opportunities and challenges do vendors face in the Gene Therapy industry?

➠ Which product types, applications, or end-users are driving market growth, and what is their market share?

➠ What are the major growth drivers and restraints of the Gene Therapy market?

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