Restore Vision and Forge Biologics Partner to Advance RV-001 Gene Therapy for Retinitis Pigmentosa Through Clinical Development
Restore Vision, Inc., a clinical-stage biotechnology company focused on developing innovative gene therapies for retinal diseases, has announced a strategic development and manufacturing partnership with Forge Biologics, a leading contract development and manufacturing organization (CDMO) specializing in gene therapies and a member of Ajinomoto Bio-Pharma Services. The collaboration is designed to support the continued advancement of RV-001, Restore Vision’s investigational adeno-associated virus (AAV)-based optogenetic gene therapy for patients living with retinitis pigmentosa (RP).
Under the agreement, Forge Biologics will provide comprehensive development and manufacturing services for RV-001, including AAV process development, current Good Manufacturing Practices (cGMP) manufacturing, analytical method development, stability testing, and regulatory consulting. These integrated services are intended to support the ongoing clinical evaluation of RV-001 while ensuring that manufacturing processes meet the rigorous quality standards required for gene therapy development.
The partnership represents an important milestone for both organizations. For Restore Vision, it strengthens the company’s manufacturing capabilities as it advances RV-001 through Phase 1/2 clinical evaluation in Japan. For Forge Biologics, the collaboration further expands its growing role in supporting global gene therapy developers and reinforces its experience in manufacturing clinical-stage gene therapies for international markets, including the Asia-Pacific (APAC) region.
Supporting the Development of Next-Generation Retinal Gene Therapies
Gene therapy has emerged as one of the most promising areas of modern medicine, offering the potential to address the underlying genetic causes of diseases rather than simply managing symptoms. In ophthalmology, gene therapies have attracted significant attention because many inherited retinal disorders are caused by well-defined genetic abnormalities that can potentially be targeted through advanced molecular technologies.
Restore Vision is dedicated to developing therapies aimed at restoring vision for individuals affected by degenerative retinal diseases. Its lead investigational program, RV-001, represents an innovative approach designed to help patients with retinitis pigmentosa, a progressive inherited retinal disorder that gradually leads to severe vision impairment and blindness.
By partnering with Forge Biologics, Restore Vision gains access to specialized expertise in gene therapy manufacturing, enabling the company to focus on advancing clinical development while relying on an experienced manufacturing partner to support production and quality assurance.
A Comprehensive Manufacturing Collaboration
The agreement encompasses a broad range of development and manufacturing activities that are essential for advancing a gene therapy from laboratory research into clinical testing.
Forge Biologics will provide:
- AAV process development
- Current Good Manufacturing Practices (cGMP) manufacturing
- Analytical development
- Stability testing
- Regulatory consultation services
Each of these components plays a critical role in ensuring that investigational gene therapies are consistently manufactured to the highest quality standards before being administered to patients.
Process development helps optimize manufacturing efficiency and product consistency, while analytical testing confirms identity, purity, potency, and overall product quality. Stability testing provides information regarding product shelf life and storage conditions, and regulatory consultation assists developers in navigating the complex regulatory requirements governing gene therapy products.
Together, these services establish a comprehensive development framework that supports clinical advancement and future regulatory submissions.
Manufacturing at Forge’s State-of-the-Art Facility
All AAV development and manufacturing activities under the collaboration are being conducted at The Hearth, Forge Biologics’ advanced manufacturing facility located in Columbus, Ohio.
The 200,000-square-foot facility has been specifically designed to support the growing needs of gene therapy developers through integrated process development, manufacturing, quality control, and regulatory services.
Following manufacturing in the United States, the finished drug product is transported to Japan for use in Restore Vision’s ongoing Phase 1/2 clinical trial.
This international manufacturing and supply chain highlights the increasingly global nature of biotechnology development, where specialized expertise and manufacturing capabilities are coordinated across multiple countries to support clinical research.
Advancing RV-001 for Retinitis Pigmentosa
The manufacturing partnership directly supports the continued clinical development of RV-001, Restore Vision’s investigational GPCR-based optogenetic AAV gene therapy.
Optogenetic therapies represent a novel therapeutic strategy for retinal diseases. Rather than replacing defective genes responsible for inherited retinal degeneration, optogenetic approaches aim to introduce light-sensitive proteins into surviving retinal cells, potentially enabling them to respond to light and restore aspects of visual function.
RV-001 utilizes an adeno-associated virus (AAV) vector to deliver genetic material designed to express a G protein-coupled receptor (GPCR)-based optogenetic construct within retinal tissue.
This innovative approach is intended to provide an alternative therapeutic option for patients who have experienced extensive photoreceptor degeneration, regardless of the specific genetic mutation responsible for their disease.
Such mutation-independent strategies have attracted increasing interest because they may be applicable to a broader population of patients affected by inherited retinal disorders.
Addressing an Unmet Need in Retinitis Pigmentosa
Retinitis pigmentosa encompasses a group of inherited retinal diseases characterized by the gradual degeneration of photoreceptor cells.
The condition typically begins with night blindness and progressive loss of peripheral vision before eventually affecting central vision in many patients.
Over time, individuals with retinitis pigmentosa may experience severe visual impairment or complete blindness.
Although significant progress has been made in understanding the genetic basis of the disease, effective treatment options remain limited for the majority of patients.
Traditional therapeutic approaches have primarily focused on slowing disease progression or managing symptoms.
Gene therapy offers the possibility of restoring visual function by targeting underlying biological mechanisms responsible for vision loss.
Restore Vision aims to address this unmet need through RV-001, providing a potential treatment option for patients who currently have few alternatives.
Supporting Clinical Evaluation in Japan
Material manufactured by Forge Biologics is currently being used in Restore Vision’s Phase 1/2 clinical trial evaluating RV-001 in Japan.
The early-stage study is designed to assess the safety and preliminary clinical activity of the investigational therapy in patients with retinitis pigmentosa.
Clinical-stage manufacturing requires strict adherence to regulatory standards to ensure patient safety and product consistency throughout the study.
By providing high-quality clinical trial material, Forge enables Restore Vision to maintain momentum as it progresses through human clinical evaluation.
The collaboration also demonstrates Forge’s ability to support multinational development programs requiring coordinated manufacturing and international product distribution.
Expanding Forge’s Presence in the Asia-Pacific Region
The Restore Vision collaboration further strengthens Forge Biologics’ growing portfolio of international partnerships.
By manufacturing product for clinical evaluation in Japan, Forge continues expanding its experience supporting gene therapy developers throughout the Asia-Pacific region.
As more biotechnology companies pursue global clinical development strategies, contract manufacturers capable of supporting multinational regulatory and manufacturing requirements have become increasingly valuable.
Forge’s integrated service model enables developers to streamline manufacturing while maintaining compliance with international quality standards.
This capability has positioned the company as a strategic partner for gene therapy innovators seeking reliable manufacturing support across multiple geographic regions.
Forge Highlights Commitment to Gene Therapy Innovation
John Maslowski, Chief Executive Officer of Forge Biologics, emphasized the company’s commitment to supporting gene therapy developers worldwide.
He noted that Forge provides integrated development, manufacturing, and regulatory expertise designed to help biotechnology companies navigate the unique challenges associated with gene therapy production.
According to Maslowski, the partnership with Restore Vision reflects Forge’s broader mission of enabling developers to advance innovative treatments while overcoming the complexities inherent in manufacturing advanced genetic medicines.
He also highlighted the shared objective of expanding treatment options for patients through successful collaboration between developers and manufacturing partners.
Restore Vision Reinforces Its Mission
Dr. Yusaku Katada, Chief Executive Officer of Restore Vision, described the partnership as an important step toward advancing the company’s mission of restoring vision for individuals living with retinitis pigmentosa.
He stated that developing a new visual restoration therapy remains central to the company’s long-term objectives.
Katada also expressed appreciation for Forge Biologics’ expertise in AAV development, manufacturing, and regulatory support, noting that these capabilities will play an important role as RV-001 progresses through Phase 1/2 clinical evaluation in Japan.
The collaboration allows Restore Vision to combine its scientific innovation with Forge’s specialized manufacturing experience, creating a strong foundation for continued clinical advancement.
Growing Demand for Specialized Gene Therapy Manufacturing
As the number of gene therapy programs entering clinical development continues to increase globally, demand for specialized manufacturing partners has grown significantly.
Unlike traditional pharmaceutical manufacturing, gene therapies require sophisticated production processes, highly controlled manufacturing environments, advanced analytical methods, and extensive regulatory oversight.
Contract development and manufacturing organizations such as Forge Biologics have become increasingly important in helping biotechnology companies efficiently translate scientific discoveries into clinical-stage products.
These partnerships allow emerging biotechnology companies to access world-class manufacturing infrastructure without the need to build extensive internal production capabilities.
The collaboration between Restore Vision and Forge Biologics marks an important milestone in the advancement of RV-001, an investigational optogenetic AAV gene therapy designed for patients with retinitis pigmentosa. By combining Restore Vision’s expertise in retinal gene therapy development with Forge’s integrated manufacturing, analytical, and regulatory capabilities, the partnership establishes a comprehensive framework for supporting the continued clinical evaluation of this promising therapeutic candidate.
As RV-001 progresses through its Phase 1/2 clinical trial in Japan, both organizations remain focused on advancing a potential new treatment option for individuals affected by inherited retinal degeneration. The partnership also reflects the growing importance of global collaboration in gene therapy development, where specialized scientific innovation, advanced manufacturing technologies, and international regulatory expertise come together to accelerate the delivery of next-generation therapies for patients with significant unmet medical needs.
About RV-001
RV-001 is Restore Vision’s lead investigational gene therapy product candidate, using adeno-associated virus (AAV) vectors with a proprietary gene encoding “Chimeric Rhodopsin,” which is a protein-based optical sensor. It is being developed as a treatment designed to restore vision in patients blinded by photoreceptor loss. Restore Vision takes a simple and minimally invasive intravitreal injection approach to deliver the therapeutic gene, which is intended to express Chimeric Rhodopsin in the remaining retinal interneurons and restore visual function.
RV-001 is currently being evaluated in a Phase 1/2 clinical trial for gene-agnostic retinitis pigmentosa in Japan. This is the world’s first clinical trial of an optogenetic gene therapy using Chimeric Rhodopsin. RV-001 is an investigational product and has not been approved by any regulatory authority. Its safety and efficacy have not been established.
About Restore Vision Inc.
Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide. To learn more, visit www.restore-vis.com.
About Forge Biologics
Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity.
Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.