Giant Axonal Neuropathy (GAN) is a rare, inherited neurodegenerative disorder characterized by progressive nerve damage due to mutations in the GAN gene, leading to impaired intracellular transport within neurons. GAN market offerings primarily comprise gene replacement therapies, viral vectors, and small-molecule modulators designed to correct or compensate for the defective gigaxonin protein. These products leverage adeno-associated virus (AAV) platforms to deliver functional copies of the GAN gene directly to affected cells, reducing axonal swellings and restoring normal neural function. Key advantages include targeted delivery to peripheral and central nervous system tissues, long-term transgene expression, and minimized frequency of dosing compared to conventional therapies.

The urgent need for effective treatments is underscored by the disease’s early childhood onset, rapid progression, and current lack of curative options. Additionally, orphan drug incentives, accelerated regulatory pathways, and growing investment in rare-disease research are fostering a supportive environment for Giant Axonal Neuropathy (GAN) Market therapy development. As clinical data continue to validate safety and efficacy, patient advocacy groups are collaborating with biotech firms to expedite trial enrollment and expand access programs.

According to CoherentMI, The giant axonal neuropathy (GAN) Market is estimated to be valued at USD 126.4 Mn in 2025 and is expected to reach USD 186.3 Mn by 2032, growing at a compound annual growth rate (CAGR) of 5.7% from 2025 to 2032.

Key Takeaways

Key players operating in the Giant Axonal Neuropathy (GAN) Market are:

-Applied Genetic Technologies Corporation (AGTC)

-Audentes Therapeutics (acquired by Astellas Pharma)

-Passage Bio

-Voyager Therapeutics

AGTC is advancing AAV-based payloads with enhanced tropism for neuronal tissue, while Audentes Therapeutics utilizes proprietary capsid engineering to improve blood–brain barrier penetration. Passage Bio focuses on scalable manufacturing processes to ensure consistent vector quality and supply, and Voyager Therapeutics is exploring novel AAV serotypes to optimize intrathecal delivery. Each of these companies has filed orphan drug applications and secured breakthrough therapy designations, underlining their commitment to rare-disease innovation. Collaborative research agreements with academic centers and contract development and manufacturing organizations (CDMOs) further bolster their competitive positioning.

Growing demand for GAN therapies is driven by increasing disease awareness, expanded genetic screening programs, and proactive newborn testing initiatives. Patient advocacy organizations are launching global registries that facilitate early diagnosis and real‐time tracking of clinical progression. These efforts accelerate trial recruitment and yield high‐quality natural history data essential for endpoint selection. Meanwhile, clinicians are calling for standardized treatment protocols and long‐term safety assessments, which have galvanized research funding from both public agencies and private foundations. As more families seek curative options, prescribers are increasingly considering investigational gene therapies over supportive care alone, fueling market uptake.

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‣ Get this Report in Japanese Language: 巨大軸索性神経障害（GAN）市場

‣ Get this Report in Korean Language: 거대축삭신경병증(GAN)시장