- Program brings together research from Steven Gray, Ph.D., and his lab at UT Southwestern with Forge’s development and manufacturing expertise to advance an AAV gene therapy for patients with TECPR2
BOCA RATON, Fla. and COLUMBUS, Ohio – September 29, 2026 – The TECPR2 Research Foundation, a nonprofit organization established to advance research and development of a treatment for patients with TECPR2, 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 advance an AAV gene therapy for patients with TECPR2, an ultra-rare neurodegenerative genetic disease.
Under the partnership, Forge is providing process development, analytical development and qualification, and manufacturing services, including the manufacture and release of material for toxicology studies, to support the program’s continued advancement. The program is leveraging Forge’s proprietary FUEL™ platform, including its HEK293 suspension Ignition Cells™, pEMBR™ 2.0 adenovirus helper plasmid, rep/cap plasmids, and program-specific optimization packages, designed to drive manufacturing efficiencies.
“For our families, this has never simply been a research program. It began with a few children and a commitment to do everything we could to create a path forward for everyone living with TECPR2,” said David Ogman, co-founder of the TECPR2 Research Foundation. “That mission has brought together an incredible group of scientists and partners who believe, as we do, that children with ultra-rare diseases deserve the same opportunity for scientific progress as anyone else.” “Forge shares that sense of purpose, and we are grateful to have their team and expertise behind us as we take these important steps toward a life-changing treatment,” said Michael Kaplan, M.D., co-founder of the TECPR2 Research Foundation.
TECPR2 is an ultra-rare genetic disorder associated with developmental delays and progressive neurological complications. TECPR2 plays an important role in autophagy, the cellular process responsible for degrading and recycling damaged or unnecessary cellular components. The investigational gene therapy is designed to use an AAV vector to deliver a functional copy of the TECPR2 gene, with the goal of addressing the underlying genetic cause of the disease.
The gene therapy program is being advanced by a research team led by AAV gene therapy pioneer Steven Gray, Ph.D., Director of the Gene Therapy Program at UT Southwestern Medical Center, together with Xin Chen, M.D., Ph.D., Assistant Professor at UT Southwestern. Their research is focused on advancing the TECPR2 Research Foundation’s AAV gene therapy toward clinical development.
“At the heart of this partnership is a shared belief in what gene therapy can make possible for patients and families,” said John Maslowski, president and chief executive officer of Forge. “When we developed our FUEL™ platform, we set out to improve manufacturing efficiency so treatments could reach more patients, with the needs of both larger patient populations and ultra-rare diseases in mind. For programs like TECPR2, making the most of every manufacturing run is critical. We are honored to bring our manufacturing technology and expertise to a program that is so deeply personal to the TECPR2 community.”
About TECPR2
TECPR2-related disorder is an ultra-rare genetic neurodevelopmental and neurodegenerative condition caused by pathogenic variants in the TECPR2 gene. TECPR2 plays an important role in autophagy, a cellular process involved in the degradation and recycling of cellular components. The disease can cause developmental delays and progressive neurological complications, including respiratory dysfunction. The investigational TECPR2 gene therapy program uses an AAV vector to deliver a functional copy of the TECPR2 gene and is being developed with the goal of addressing the underlying genetic cause of the disease.
About the TECPR2 Research Foundation
The TECPR2 Research Foundation is a nonprofit founded by physicians and families worldwide with a single mission: to advance research and curative treatments for TECPR2, a rare and fatal childhood disease also known as SPG49 or HSAN9. The foundation unites families, leading clinicians, geneticists, and biotech partners to accelerate first of its kind therapeutics for children living with the disease. To learn more, visit www.tecpr2.org.
About Forge Biologics
Forge Biologics is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by helping bring them from concept to reality. Headquartered in Columbus, Ohio, Forge operates the Hearth, a 200,000-square-foot facility with 20 custom-designed cGMP suites and 20,000 L of bioreactor capacity. Forge provides end-to-end AAV development and manufacturing services from research scale through commercial production, leveraging its proprietary FUEL™ platform designed to get more doses from every run. Forge is part of Ajinomoto Bio-Pharma Services, a global network of specialized CDMOs within the Ajinomoto Group. To learn more, visit www.forgebiologics.com.
Media Inquiries
TECPR2 Research Foundation:
Michael Kaplan, M.D.
Co-Founder, Executive Director
info@tecpr2.org
Forge Biologics:
Marina Corleto
Associate Director, Marketing & Communications
media@forgebiologics.com
Client Development:
Taleen Barsoumian
Senior Vice President, Commercial
CD@forgebiologics.com
- 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
- Partnership marks a critical step toward clinical development of an AAV gene therapy using a base editing approach designed to target theapproach designed to target the genetic cause of Hutchinson-Gilford Progeria Syndrome
- Forge Biologics will provide process development and manufacturing expertise to support gene therapy advancement led by world-renowned researchers Leslie Gordon, David Liu, and Francis Collins
PEABODY, MA and COLUMBUS, Ohio – March 3, 2026 – The Progeria Research Foundation (“PRF”), a nonprofit research organization dedicated to developing treatments and the cure for Hutchinson-Gilford Progeria Syndrome (“Progeria”), and Forge Biologics, (“Forge”), a leading manufacturer of gene therapies and member of the Ajinomoto Bio-Pharma Services group, today announced a manufacturing agreement to support the development and manufacturing of SamPro-2, PRF’s investigational gene therapy for children and young adults living with Progeria, an ultrarare and fatal genetic disease characterized by rapid aging.
The manufacturing agreement brings together PRF’s decades-long, research-driven Progeria program with Forge’s integrated gene therapy capabilities including process development, cGMP manufacturing, FUEL™ platform technologies, and regulatory consultation. Forge will provide manufacturing services for Investigative New Drug (IND)-enabling studies with SamPro-2, a gene therapy that uses adeno-associated virus (AAV), a commonly utilized delivery vehicle in gene therapy, to deliver a base editing approach designed to correct the single DNA base mutation in the lamin A gene that causes Progeria.
“The era of Progeria gene therapy has arrived. Our hope is that SamPro-2 will give children and young adults with Progeria the longer, healthier lives they deserve,” said Leslie Gordon, M.D., Ph.D., co-founder and medical director of PRF and the mother of Sam Berns who had Progeria. “We are extremely grateful to be working with Forge Biologics, whose manufacturing expertise is essential to move this work from the laboratory towards clinical trials.”
PRF and its collaborators, collectively known as the Progeria Gene Team, have created SamPro-2, a CRISPR-based gene editing strategy designed to permanently correct the Progeria genetic mutation at its source. The effort is led by Dr. Gordon, a leading Progeria expert and clinical trialist; David R. Liu, Ph.D., Richard Merkin Professor and Director of the Merkin Institute for Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard, whose laboratory has been an international leader in the development of base editing technology; Francis S. Collins, M.D., Ph.D., Senior Research Advisor to the Progeria Research Foundation; and the late Sammy Basso, M.S., former scientist, advocate, and enduring inspiration whose legacy continues to guide this work.
“Behind every program like this are patients and families who have waited a long time for progress,” said John Maslowski, president and chief executive officer of Forge Biologics. “The Progeria Research Foundation and its Gene Team have shown extraordinary dedication to advancing this science, and we are honored to partner with them. At Forge, we bring that same level of care, expertise, and technical rigor to our manufacturing work as we help advance this program for patients.”
About SamPro-2
SamPro-2 is an investigational in vivo gene-editing therapy being advanced by The Progeria Research Foundation (PRF) as part of its Path to Cure Progeria initiative. SamPro-2 is designed to correct the underlying genetic cause of Hutchinson-Gilford Progeria Syndrome (Progeria) using precision base-editing technology, packaged in an AAV9 capsid, to correct the single DNA letter change that leads to production of the toxic protein progerin.
About The Progeria Research Foundation
The Progeria Research Foundation (PRF) is the driving force behind the global effort to understand, treat and ultimately cure Hutchinson-Gilford Progeria Syndrome (Progeria), a rare and fatal genetic disease that causes rapid aging in children. Founded by the family of Sam Berns after his diagnosis in 1999, PRF has enabled or led every major scientific breakthrough in the field, from discovery of the gene that causes the disease to the first FDA-approved treatment, lonafarnib, to the advancement of gene-editing approaches now in development. Through rigorous science, global research infrastructure and close partnership with the worldwide patient community, PRF is advancing next-generation therapies, expanding diagnosis and care through its Find the Children initiative, and leading the Path to Cure Progeria program to determine whether a one-time gene-editing therapy can offer a durable, potentially curative treatment. For more information and to support PRF’s mission, please visit www.progeriaresearch.org.
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
The Progeria Research Foundation:
Tracy Lessor
Director of Communications
tlessor@progeriaresearch.org
Forge Biologics:
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
- 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