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Neurophth and AAVnerGene Enter Strategic AAV Capsids Partnership for Next-Generation Ophthalmic Gene Therapy

Published: Jan 19, 2021

  • Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy

  • Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost.

Wu HAN, China and NEWARK, DE., Jan. 19, 2021 /PRNewswire/ -- Neurophth Therapeutics, Inc., a subsidiary of Wuhan Neurophth Biotechnology Ltd. gene therapy company (hereafter Neurophth), and AAVnerGene Inc., a specializing AAV technology company, today announced a strategic partnership to provide Neurophth with worldwide rights to mutually select adeno-associated virus (AAV) capsids for the next generation ophthalmic gene therapy.

AAVnerGene's Tissue-specific, Highly-transductive and Expressive New AAVs (ATHENA) screening platform can efficiently select the best AAV vector for each cell type in a high-throughput (HTP) manner. Major challenges in AAV-based gene therapy are the transduction efficiency, possible immunity to the capsid and complex manufacturing processes. The ability to identify suitable next generation AAV vectors that could overcome the limitations of earlier generation AAV vectors is critical to a patient for achieving sufficient therapeutic expression of the transferred gene in the lowest dose through common procedure such as intravitreal (IVT) injection with reasonable cost.

"AAVnerGene's proprietary technology may create capsid libraries derived from artificial intelligence machine learning, DNA shuffling or directed evolution allowing a significant increase in AAV genetic payload capacity, production, and transduction with the ability to penetrate through the inner limiting membrane of the retina, thus potentially enhance the overall transduction efficiency of capsid library-derived AAV vectors," said Alvin Luk, Ph.D., M.B.A., Chief Executive Officer of Neurophth. "Importantly, if proven successful, the administration of the selected AAVnerGene capsid variant(s) in gene therapy may enable repeated dosing AAVs in both adults and pediatric patients, potentially improving the clinical efficacy at a lower vector dose with better penetration of the barriers in eyes using a safer and less invasive procedure such as intravitreal injection which lower the risk of immune response to the capsid."

"AAV is the most widely-used vector in gene therapy, however, its efficacy and cost will need significant improvement. Suitable AAV plays a vital role to achieve the success of a gene therapy. Our ATHENA platform is designed to select the optimal AAV vector for different cells and diseases," said Daozhan Yu, Ph.D., Chief Executive Officer of AAVnerGene. "We are proud to partner with Neurophth, a recognized gene therapy leader, to accelerate the application of our AAV capsid libraries in gene therapy. Both parties aim to build a strategic partnership for long term success which will bring accessible AAV gene therapy treatment to patients around the world."

Subject to the terms of the agreement, Neurophth will make an initial cash payment to AAVnerGene to test ~100 AAV capsids. Within 6-12 months upon receiving the AAV capsids, Neurophth is responsible for completing the preclinical studies on these capsids. Thereafter, both companies will jointly evaluate the capsids and selected the appropriate ones for further development. Upon the selection of the appropriate capsids, Neurophth has agreed to pay AAVnerGene additional cash and/or to potentially purchase AAVnerGene's common stock being based on Neurophth's achievement of specified development, regulatory and commercial milestones. In addition, Neurophth will pay AAVnerGene royalties for the sales of any resulting commercialized gene therapy.

The terms of this agreement do not apply to Neurophth's ongoing investigational development programs in Leber hereditary optic neuropathy (LHON), including NR082 (NFS-01 program) for the treatment of ND4-mutated LHON and ND1-mutated LHON. This agreement does not impact Neurophth Therapeutics' ongoing research in glaucoma, wet age-related macular degeneration (wAMD) and diabetic macular edema (DME).

About Neurophth

Neurophth is China's first gene therapy company in ophthalmic diseases. Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and US, Neurophth, a fully integrated company, is striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. Our AAV validated platform which has been published in Nature - Scientific Reports, Ophthalmology, and EBioMedicine, successfully delivered proof-of-concept data with investigational gene therapies in the retina. Our most advanced investigational candidate, NR082 (rAAV2-ND4), in development for the treatment of ND4-mutated LHON, has received orphan drug designations in the U.S. The pipeline also includes ND1-mutated LHON, autosomal dominant optic atrophy, glaucoma, wAMD/DME, and other preclinical candidates. Neurophth has initiated the scaling up in-house process in single-use manufacturing technologies to support future commercial demand at the Suzhou facility. To learn more about us and our growing pipeline, visit

About AAVnerGene

AAVnerGene Inc. is an innovative company leveraging AAV technologies to improve patients' lives through the potential of gene therapy. With over 20 years of frontline gene therapy expertise, the company develops novel AAV vectors, methods and strategies that can efficiently produce, deliver and express high quality vectors into specific cells. The company's goal is to utilize these vectors from ATHENA screening platform to enable the development of single administration of AAV gene therapy with higher expression, increased durability, reduced side effect and improved manufacturability, making it accessible to patients globally. For more information, please visit

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