- Forge’s development and manufacturing services support RV-001, Restore Vision’s gene therapy for patients with retinitis pigmentosa, in Phase 1/2 clinical evaluation in Japan
TOKYO, JAPAN and COLUMBUS, OH – July 21, 2026 – Restore Vision, Inc. (“Restore Vision”), a clinical-stage biotechnology company advancing gene therapies for retinal disorders, and Forge Biologics (“Forge”), a leading manufacturer of gene therapies and member of Ajinomoto Bio-Pharma Services, today announced a development and manufacturing partnership to support RV-001, Restore Vision’s investigational GPCR-based optogenetic AAV gene therapy for patients with retinitis pigmentosa.
Through this collaboration, Forge is providing Restore Vision with AAV process development, current Good Manufacturing Practices (cGMP) manufacturing, analytical development, stability testing, and regulatory consultation services. The AAV development and manufacturing activities performed by Forge under this collaboration take place at the Hearth, Forge’s 200,000-square-foot facility in Columbus, Ohio, and the drug product is transported to Japan for clinical trial supply.
“Forge is proud to support gene therapy developers around the world with integrated development, manufacturing, and regulatory expertise,” said John Maslowski, Chief Executive Officer of Forge Biologics. “Our partnership with Restore Vision reflects our commitment to helping developers navigate the complexities of gene therapy manufacturing, with the shared goal of enabling treatment options for patients.”
Material manufactured at Forge for Restore Vision is being used to evaluate RV-001 in a Phase 1/2 clinical trial in Japan, further expanding Forge’s experience supporting clinical-stage gene therapy programs in the Asia-Pacific (APAC) region.
“Providing a new visual restoration treatment option for patients living with retinitis pigmentosa is central to our mission,” said Yusaku Katada, M.D., Ph.D., Chief Executive Officer of Restore Vision. “We are pleased to collaborate with Forge Biologics and value their expertise in AAV development, manufacturing, and regulatory support as we advance RV-001 through Phase 1/2 clinical evaluation in Japan.”
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.
Media Inquiries
Restore Vision:
Hikaru Miyazaki
Chief Operating Officer
contact@restore-vis.com
Forge Biologics:
Media
Marina Corleto
Associate Director, Marketing & Communications
media@forgebiologics.com
Client Development
Taleen Barsoumian
Senior Vice President, Client Development
CD@forgebiologics.com
- Skylark Bio is leveraging Forge’s AAV FUEL™ platform technologies and cGMP manufacturing services to advance its gene therapy for patients with genetic hearing loss
CAMBRIDGE, MA and COLUMBUS, OH – June 16, 2026 – Skylark Bio (“Skylark”), a clinical-stage biotechnology company developing targeted genetic medicines for hearing loss, and Forge Biologics (“Forge”), a leading manufacturer of gene therapies and member of Ajinomoto Bio-Pharma Services, today announced a strategic AAV development and cGMP manufacturing partnership.
Under the partnership, Forge is providing Skylark with process development, current good manufacturing practice (cGMP) manufacturing, and analytical development services. Skylark is also leveraging Forge’s proprietary FUEL™ platform technologies, including its HEK293 suspension Ignition Cells™ and pEMBR™ 2.0 adenovirus helper plasmid. All development and manufacturing activities are being conducted at the Hearth, Forge’s 200,000-square-foot gene therapy development and manufacturing facility in Columbus, Ohio.
“As we enter the clinic with our lead program, Skylark needs a manufacturing partner who shares our commitment to excellence,” said Jodi Cook, Ph.D., President and Chief Executive Officer of Skylark. “Forge provides the partnership we need to confidently advance our research into clinical development. Their end-to-end AAV gene therapy manufacturing capabilities provide exactly the foundation we’re looking for, and they’ve integrated seamlessly with our team to help ensure our therapies are manufactured with the rigor and care that patients deserve.”
Skylark’s lead program, SKY-GJB2, is currently being evaluated in a Phase 1/2 clinical trial for GJB2-related hearing loss, the leading genetic cause of inherited hearing loss worldwide. The investigational AAV gene therapy is designed to deliver a functional copy of the GJB2 gene directly to cells in the inner ear affected by disease-causing mutations, with the potential to address the underlying genetic cause of hearing loss. SKY-GJB2 leverages Skylark’s proprietary capsid platform, which is engineered for targeted, low-dose delivery and durable gene expression.
“At the heart of our collaboration with Skylark is a shared mission to bring meaningful treatment options to patients,” said John Maslowski, President and Chief Executive Officer of Forge. “This partnership reflects our focus on improving how gene therapies are made so more promising medicines can reach those who need them. By combining our platform technologies with manufacturing expertise, we are proud to support Skylark as it advances its gene therapy program through clinical development.”
About Skylark Bio
Skylark Bio is a clinical-stage genetic medicine company developing precision, locally delivered gene therapies for monogenic diseases, with the potential to meaningfully improve patient outcomes and quality of life. The company’s lead program, SKY‑GJB2, is in a Phase 1/2 first‑in‑human clinical trial for GJB2‑related hearing loss—the leading cause of inherited hearing loss worldwide. A second program targeting SLC26A4‑related hearing loss (Pendrin) is advancing through IND‑enabling studies. Built on a proprietary capsid engineered for broad transduction, Skylark’s platform enables targeted, micro‑dose delivery with cell‑specific expression control and the potential for durable benefit in non‑dividing cells of interest. The company is led by a team with deep expertise in gene therapy and supported by world‑class advisors as it expands its platform into additional hearing and CNS indications. For more information, visit Skylarkbio.com and follow us on LinkedIn.
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-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.
Media Inquiries
Skylark Bio:
Susan Sharpe
Linnden Communications
susan@linndencom.com
Forge Biologics:
Media
Marina Corleto
Associate Director, Marketing & Communications
media@forgebiologics.com
Client Development
Taleen Barsoumian
Senior Vice President, Client Development
CD@forgebiologics.com
- Forge’s FUEL™ platform and cGMP manufacturing services support the production of AAV for Ascidian’s ACDN-01, the most advanced genetic therapy targeting the underlying cause of Stargardt disease
- Collaboration secures clinical supply needed to advance ACDN-01 into late-stage studies
BOSTON, MA and COLUMBUS, OH – January 7, 2026 – Ascidian Therapeutics (“Ascidian”), a biotechnology company seeking to treat human diseases by rewriting RNA, and Forge Biologics (“Forge”), a leading manufacturer of gene therapies and member of the Ajinomoto Bio-Pharma Services group, today announced a strategic partnership to advance the development and manufacturing of ACDN-01, Ascidian’s lead retinal program for the treatment of Stargardt disease.
Through this collaboration, Forge is providing Ascidian with process and analytical development services, toxicology, and current Good Manufacturing Practice (cGMP) manufacturing. Ascidian is utilizing Forge’s proprietary FUEL™ technologies, including HEK293 suspension Ignition Cells™ and pEMBR™ 2.0 adenovirus helper plasmid, along with program-specific optimizations designed to drive manufacturing efficiencies. All development and manufacturing activities take place at the Hearth, Forge’s 200,000-square-foot gene therapy development and manufacturing facility in Columbus, Ohio.
“We are proud to partner with Ascidian Therapeutics on this program, which reflects our shared commitment to bringing together scientific and manufacturing innovation to support patients,” said John Maslowski, President and Chief Executive Officer of Forge. “Forge’s FUEL™ platform technologies and manufacturing capabilities were built to enable partners like Ascidian as they advance their work through clinical development and beyond.”
Ascidian is currently evaluating ACDN-01—the most advanced genetic therapy targeting the underlying cause of Stargardt disease—in the Phase 1/2 STELLAR dose-escalation trial. ACDN-01 uses a single AAV vector to perform in vivo RNA exon editing, restoring full-length ABCA4 protein that is deficient in Stargardt patients. This first-in-class approach has already demonstrated durable, efficient editing in both non-human primate and human retinal models.
“ACDN-01 represents a fundamentally new approach to treating diseases driven by large, complex genes like ABCA4 which have historically been difficult to address with traditional gene therapies,” said Michael Ehlers, M.D., Ph.D., President and Chief Executive Officer of Ascidian. “Partnering with Forge Biologics equips us with deep AAV expertise, scalable manufacturing capabilities, and significant capacity, enabling us to rapidly advance ACDN-01 into late-stage clinical development.”
About Ascidian Therapeutics
Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs. To learn more about Ascidian, visit www.ascidian.com. To learn more about the STELLAR clinical trial, visit www.StellarStargardtTrial.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.
Media Inquiries
Ascidian Therapeutics:
Heather Shea
heather.shea@catalyticagency.com
Forge Biologics:
Media
Marina Corleto
Associate Director, Marketing & Communications
media@forgebiologics.com
Client Development
Taleen Barsoumian
Senior Vice President, Client Development
CD@forgebiologics.com
- FUEL™ platform can achieve a 2-6x increase in productivity compared to industry standard
- New innovative platform technologies include pEMBR 2.0™ Ad helper and modified rep/cap plasmids
- Data on FUEL™ platform will be presented at Cell & Gene Meeting on the Mesa
COLUMBUS, OHIO October 2, 2024 – Forge Biologics, a member of Ajinomoto Bio-Pharma Services and leading manufacturer of genetic medicines, today announced the launch of its FUEL™ manufacturing platform to provide AAV gene therapy developers a more efficient and accelerated foundation for manufacturing as they advance new programs and target a broader range of diseases.
The FUEL™ (Foundation for Unleashing Excellence in Life-Changing Therapies) platform introduces several new technical advancements, including Forge’s pEMBR 2.0™ Ad helper plasmid which is one of the smallest commercially available at 8.9kb, offering an enhanced safety profile and increased manufacturing efficiency. The platform also features new modified rep/cap plasmids, whereby a client-specific capsid sequence is incorporated into Forge’s proprietary backbone plasmid. Forge’s trusted HEK293 suspension Ignition™ cell line remains a vital component of the platform. Together, these proprietary technologies, combined with Forge’s proven manufacturing processes and unique optimization packages provide a faster, potentially safer, and more efficient foundation for AAV production.
“As a CDMO, we prioritize continuous innovation to deliver effective solutions for our groundbreaking clients who are focused on advancing their programs from the discovery phase to first-in-human clinical trials and beyond,” said John Maslowski, President & CEO of Forge. “The FUEL™ platform represents years of extensive R&D on our innovative technologies and builds upon our well-established manufacturing processes with a deep understanding of the need for product-specific flexibility with numerous optimization packages.”
“I’m incredibly proud of our team’s exceptional collaboration that has brought us to this point with the FUEL™ platform and the productivity gains we’re seeing,” said David Dismuke, Ph.D., Chief Technical Officer of Forge. “From molecular development’s research for our new pEMBR 2.0™ Ad helper design, to our process development team’s repeatable high-titer manufacturing processes, Forge remains focused on driving manufacturing excellence for our clients and the industry as it continues to expand and reach broader patient populations.”
Forge will launch the FUEL™ platform and highlight its key benefits during a company presentation by Mr. Maslowski on October 8, 2024, at 3:15 p.m. during the Alliance for Regenerative Medicine’s Cell & Gene Meeting on the Mesa conference in Phoenix, Arizona. For more information, please visit https://meetingonthemesa.com/.
About Forge Biologics
Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, 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.
Media Inquiries
Marina Corleto
Associate Director, Marketing and Communications
media@forgebiologics.com
COLUMBUS, OHIO September 9, 2024 – Forge Biologics, a member of Ajinomoto Bio-Pharma Services and leading manufacturer of genetic medicines, today announced it will be the adeno-associated virus (AAV) development and manufacturing partner for the Muscular Dystrophy Association’s (MDA) Kickstart Program.
MDA is the leading nonprofit focused on accelerating research, advancing care, and advocating for the support of people living with muscular dystrophy, ALS, and related neuromuscular diseases. The MDA Kickstart Program is a new research program developed as a strategy to lower commercial barriers and de-risk development of gene therapies for ultra-rare neuromuscular diseases.
“Collaborating with the MDA embodies our mission at Forge to help deliver potentially life-changing treatments to patients suffering from rare disease,” said John Maslowski, Chief Commercial Officer at Forge Biologics. “We are excited to support the MDA Kickstart Program and help advance these critical projects to the next stage of development.”
Forge will provide process and analytical development manufacturing services, and the partnership will leverage Forge’s platform process, including its proprietary HEK293 suspension Ignition Cells™ and pEMBR™ adenovirus helper plasmid. All development and manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy manufacturing facility in Columbus, Ohio.
“Forge has proven to be a trusted partner to gene therapy innovators, advancing many critical programs for rare diseases. We look forward to embarking on this manufacturing partnership, leveraging their deep expertise and robust platform to propel our Kickstart Program’s goal of accelerating the delivery of treatments and cures to patients with ultra-rare neuromuscular diseases,” said Sharon Hesterlee, Ph.D., Chief Research Officer at the Muscular Dystrophy Association.
About Forge Biologics
Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, 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 200,000L of manufacturing 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.
Media Inquiries
Marina Corleto
Associate Director, Marketing and Communications
mcorleto@forgebiologics.com
- Research findings indicate a path to the development of potentially safer adeno-associated virus (AAV) gene therapies and provide additional avenues for improving AAV manufacturability for increasing yields
- The discovery was published as a peer-reviewed scientific article in a leading research journal Human Gene Therapy
COLUMBUS, Ohio December 12, 2023 –Forge Biologics (Forge), a leading manufacturer of genetic medicines, announced today the peer-reviewed publication of, “A novel role for the adenovirus L4 region 22K and 33K proteins in adeno-associated virus production,” in the research journal Human Gene Therapy. The article was authored by a team of scientists at Forge led by David Dismuke, Ph.D., Chief Technical Officer, Linas Padegimas, Ph.D., Molecular Development Senior Director, and Angela Adsero, Ph.D., Molecular Development Scientist II.
Specific adenoviral genes are required for AAV production. However, a novel, undiscovered gene in particular has been overlooked because of its shared DNA sequence with a gene regulatory region that determines when or how much protein is made from a gene. By using molecular techniques to decouple the dual-purpose nature of this sequence, the Forge scientific team demonstrated that the L4 region 22K protein is an additional requirement for AAV vector production that had previously gone unnoticed. The study also suggests that the L4 region 33K protein is important for increasing AAV production.
“Forge is committed to improving gene therapy manufacturing through scientific innovation. The discovery of these vital AAV production requirements provide the potential for a more targeted and efficient production strategy, while also strengthening our IP portfolio,” said David Dismuke, Ph.D., Chief Technical Officer at Forge. “I am exceptionally proud of the Forge team for their hard work in contributing to our understanding of the essential elements of AAV production so that we can keep advancing the field of gene therapy.”
The article’s lead author was Angela Adsero, Ph.D. Contributing authors include the following Forge scientists: Brendan Chestnut, M.Sc., Sara Shahnejat-Bushehri, Ph.D., Lalita Sasnoor, Ph.D., Travis McMurphy, Ph.D., Michael Swenor, Ryan Pasquino, Arun Pradhan, Ph.D., Victor Hernandez, Ph.D., Linas Padegimas, Ph.D., and David Dismuke, Ph.D. The full research article can be accessed through open access here: https://www.liebertpub.com/doi/epdf/10.1089/hum.2023.146
“This interesting finding may significantly impact gene therapy manufacturing and that translates to improved and potentially life-transforming genetic medicines for millions of patients suffering from genetic diseases worldwide,” said Robert Kotin, Ph.D., a leading voice in AAV and gene therapy manufacturing and a member of Forge’s Scientific and Manufacturing Advisory Board. “The Forge team has deep roots in developing genetic medicines and in vector manufacturing, as evidenced by these findings.”
About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company focused on enabling access to life-changing gene therapies. Forge was founded in 2020 and is headquartered in the cell and gene therapy hub of Columbus, Ohio. Its 200,000 square foot facility, the Hearth, is dedicated to AAV manufacturing with 20 custom-designed cGMP suites. Offerings include scalable, end-to-end manufacturing services including process and analytical development, cGMP viral vector manufacturing, final fill, plasmid DNA manufacturing, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. Forge aims to accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.
Media Inquiries
Marina Corleto
Associate Director, Marketing and Communications
media@forgebiologics.com
Client Development
Taleen Barsoumian
Vice President, Client Development
CD@forgebiologics.com