Gene Therapy IND Overview (Jan–Mar)

In the biopharmaceutical field, gene therapy has emerged as a beacon of hope for tackling complex diseases due to its revolutionary treatment concepts and potential for cures. From January to March 2025, the global gene therapy sector has continued to heat up, with several new Investigational New Drug (IND) applications approved, injecting new momentum into the development of gene therapies and bringing hope to many patients.

Solid Biosciences – SGT-212

On January 7, Solid Biosciences announced that the U.S. Food and Drug Administration (FDA) had approved the IND application for its gene therapy SGT-212 to treat Friedreich's ataxia. It is the only full-length frataxin replacement gene therapy candidate targeting both the central nervous system and cardiac manifestations of Friedreich's ataxia—a hereditary, degenerative multisystem disorder caused by a frataxin gene defect, resulting in progressive nervous system damage, motor issues, and cardiac dysfunction, with heart complications being the leading cause of death. SGT-212 is a recombinant gene replacement therapy based on AAV, delivered via dual administration routes (intravenous infusion and direct injection into the dentate nucleus of the cerebellum) to increase therapeutic frataxin levels in cerebellar and myocardial cells, aiming to repair mitochondrial dysfunction and address neurological and cardiac symptoms.

Hengrui Pharma Subsidiary – RGL-2201

On January 16, RGL-2201, the first dual-target AAV gene therapy candidate drug developed by Hengrui Pharma’s subsidiary Ruihongdi Pharma, received implied IND approval from the Center for Drug Evaluation (CDE) in China. It is intended to treat neovascular age-related macular degeneration (nAMD), a common blinding eye disease affecting the elderly. With a unique dual-target design, RGL-2201 aims to precisely act on lesion sites and inhibit neovascularization, potentially improving vision and offering a new treatment option for nAMD.

Belief BioMed – BBM-D101

On January 22, Belief BioMed’s AAV gene therapy candidate BBM-D101 injection received FDA IND approval for treating Duchenne muscular dystrophy (DMD). Notably, the day before, the IND was also accepted by the CDE in China. DMD is a severe hereditary muscle disorder that typically manifests in childhood and progressively worsens, severely impacting quality of life and lifespan. BBM-D101 aims to deliver a functional gene to compensate for the defective one via gene replacement therapy, bringing new therapeutic hope to DMD patients.

Bayer / AskBio – AB-1005 for Parkinson’s Disease

On February 19, AskBio, a wholly owned and independently operated gene therapy subsidiary of Bayer, announced that its gene therapy drug AB-1005 for Parkinson’s disease (PD) had received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. AB-1005 is an AAV2-based gene therapy containing a human glial cell line-derived neurotrophic factor (GDNF) transgene. It is administered via neurosurgical direct injection (MRI-guided convection-enhanced delivery) for stable, sustained expression of GDNF in targeted brain regions. The FDA's RMAT designation was based on clinical data from an open-label, non-controlled Phase 1b study, which showed good tolerability and no serious product-related adverse events. Moderate PD patients showed improvement or stability on various clinical scales, while most mild PD patients maintained overall stability. This recognition underscores AB-1005’s potential and accelerates its development.

Eg427 – EG110A

On February 21, Eg427, a biotech company focused on treating chronic neurological disorders using non-replicating HSV-1 (nrHSV-1) vector technology, announced that its lead therapy EG110A had received FDA IND approval in June 2024 and is now undergoing clinical trials. This is the first human trial of such a vector for treating sensory neuron diseases. The funding received will support Eg427’s expansion of its therapeutic pipeline using its proprietary Hermes platform, which enables precise delivery of neurotherapeutics to peripheral and central nervous systems, opening new treatment possibilities for chronic neurological diseases.

Shenji Changhua – SNUG01

On March 24, Shenji Changhua (Beijing) Biotechnology announced that its independently developed gene therapy drug SNUG01, targeting TRIM72 and indicated for amyotrophic lateral sclerosis (ALS), received FDA IND approval. This approval allows it to enter an international multi-center Phase I/IIa clinical trial to assess its safety, tolerability, and preliminary efficacy in adult ALS patients. ALS is a progressive, fatal neurodegenerative disorder affecting motor neurons, with a median survival of just 3–5 years and no current cure. Based on original research from Professor Jia Yichang’s lab at Tsinghua University, SNUG01 uses an rAAV9 vector administered via intrathecal injection to deliver the human TRIM72 gene to neurons. Preclinical studies showed significant neuroprotection, and earlier investigator-initiated trials led by Peking University Third Hospital confirmed its safety and showed promising efficacy and biomarker signals.

Baison Bio – BN-1001 Ophthalmic Injection

On March 6, Nanjing Baison Bio’s independently developed gene therapy drug BN-1001 ophthalmic injection received implied clinical trial approval from the CDE for a new indication: diabetic macular edema (DME). The product had previously received implied approval for treating neovascular (wet) age-related macular degeneration. Using an AAV-based gene therapy approach, BN-1001 is the lowest-dose product among its peers worldwide with sustained expression of anti-VEGF molecules. It holds promise for “one treatment, lifetime cure” and addresses unmet clinical needs.

Jianda Jiuzhou – GA001 Injection

As a major scientific achievement of the 2025 Zhongguancun Forum, Jianda Jiuzhou’s “gene therapy technology based on novel photosensitive proteins” will enter Phase I clinical trials this year. Their independently developed Class I biological new drug GA001 injection had its first research subject dosed in January 2024, with all 9 subjects now treated. Most showed significant vision improvement, with some able to recognize nearby letters and shapes, and no drug-related adverse reactions observed.

The approvals of gene therapy INDs from January to March fully demonstrate the sector’s vigorous development and vast potential. From rare diseases to neurological disorders, each approval marks a solid step forward in conquering major diseases.