COLUMBUS, OHIO April 30, 2024 – Forge Biologics, a member of Ajinomoto Bio-Pharma Services and leading manufacturer of genetic medicines, today announced that it will deliver nine presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27^thAnnual Meeting, taking place May 7-11, 2024, in Baltimore, Maryland. The presentations include a late-breaking oral presentation, three technical session presentations, and five scientific posters spanning Forge’s process development, molecular development, integrated regulatory services, and clinical program advancements.

“As we continue to advance how genetic medicines are manufactured, we are thrilled to present data showcasing our novel molecular findings, platform process improvements, and regulatory knowledge all centered around efficient, safe, and scalable manufacturing of AAV gene therapies,” said Timothy J. Miller, Ph.D., President and CEO of Forge. “We are also especially encouraged by a late-breaking oral presentation that our Chief Medical Officer, Dr. Maria Escolar, will be delivering that provides a clinical update on FBX-101 where all five patients with Krabbe disease receiving Forge’s gene therapy are walking—a remarkable result. Retention of gross-motor function is one of the most visible and debilitating symptoms seen in patients with Krabbe disease, so this finding is a meaningful clinical outcome.”

ORAL PRESENTATION

Title: REKLAIM, A Phase 1b Clinical Trial Using a Novel Immune Modulation Strategy for Systemic Administration of FBX-101 (AAVrh10.GALC) After Umbilical Cord Blood Transplantation for the Treatment of Infantile Krabbe Disease
Presenter: Maria Escolar, M.D., Chief Medical Officer
Date and Time: Friday, May 10, 2024, 8:00-8:15 a.m. ET
Room: Ballroom 4

SESSION PRESENTATIONS

ASGCT Workshop
Title: Preparing for CMC Success: How Early CMC Decision Drives Your Pace and Success to the Clinic
Presenter: David Dismuke, Ph.D., Chief Technical Officer
Date and Time: Tuesday, May 7, 8:25 a.m. ET
Location: Ballroom 3

Tools & Technology Forum
Title: Improving Analytical Tools for AAV Characterization
Presenter: David Dismuke, Ph.D., Chief Technical Officer
Date and Time: Wednesday, May 8, 2:15-2:30 p.m. ET
Location: Exhibit Hall Theater

Sponsored Symposium
Title: Forge’s Discovery and Modification of Genetic Elements for Enhanced Productivity and Efficacy of AAV-based Gene Therapies
Presenters:
          Angela Adsero, Ph.D., Molecular Development Scientist II
          Frank Agbogbo, Ph.D., VP of Process Development
          David Dismuke, Ph.D., Chief Technical Officer
Date and Time: Thursday, May 9, 1:30-2:00 p.m. ET
Location: Room 337-338

POSTER PRESENTATIONS

Title: Development of a Scalable Upstream Process for Suspension HEK 293 rAAV Production in Single-Use Bioreactors for Efficient Manufacturing of Gene Therapy Products
Presenter: Danielle Sexton, Senior Scientist II, Process Development
Date and Time: May 8, 12:00 p.m. ET
Poster number: 528

Title: From Benchtop to Cleanroom; Designing an Optimized Platform Purification Process for rAAV Production to Meet the Growing Demand
Presenter: Blake Gursky, Scientist II, Process Development
Date and Time: May 8, 12:00 p.m. ET
Poster number: 527

Title: The Potential of Global Regulatory Harmonization in Gene and Cell Therapy
Presenter: Claire Marasco, Associate Director, Regulatory Affairs
Date and Time: May 8, 12:00 p.m. ET
Poster number: 526

Title: Regulatory Agility: Value of CDMOs Utilizing Platform Technology
Presenter: Angela Coy, Ph.D., Senior Manager, Regulatory Affairs
Date and Time: May 8, 12:00 p.m. ET
Poster number: 905

Title: The Sixth Element: Identification and Evaluation of the Adenovirus L4 22/33K Region as a Requirement for Adeno-Associated Virus Production
Presenter: Angela Adsero, Ph.D., Molecular Development Scientist II, Forge Biologics
Date and Time: May 9, 12:00 p.m. ET
Poster number: 954

Full abstracts are available on the ASGCT Annual Meeting website, and conference participants can also access posters through the ASGCT website.

Forge Biologics will be at booth #827 in the Exhibitor Hall.

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
media@forgebiologics.com

Families and Clinician Inquiries
Maria Escolar, M.D.
Chief Medical Officer
advocacy@forgebiologics.com

Business Development  
Magdalena Tyrpien
Chief Business Officer
BD@forgebiologics.com   

• BGTC aims to accelerate the development, manufacturing, and delivery of customized or ‘bespoke’ gene therapies to millions of patients with rare diseases 
• Christopher Shilling, Senior Vice President of Regulatory Affairs and Quality, to represent Forge on BGTC Steering Committee  

COLUMBUS, Ohio – May 23, 2023 – Forge Biologics, a leading manufacturer of genetic medicines, announced today that it has joined the Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration to accelerate the delivery of promising new gene therapies to patients with rare diseases. Forge has appointed Christopher Shilling, Senior Vice President of Regulatory Affairs and Quality, to represent the Company on the BGTC’s Steering Committee.  

The BGTC is managed by the Foundation for the National Institutes of Health (FNIH) as part of the Accelerating Medicines Partnership® program, a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), biopharmaceutical and life science companies, and nonprofit and other organizations on the consortium, to collectively work towards speeding the process of gene therapy development and manufacturing for rare diseases.  

The consortium was formed to help bridge the gap that exists in the current drug development model that makes it difficult for companies to recover the costs required to develop gene therapies to treat patients with rare diseases. By creating a standardized, reusable approach that reduces up-front costs, the BGTC will help to lower development barriers, enabling meaningful advancement against a number of rare diseases.

“Forge has a shared goal with the BGTC of accelerating access of effective genetic therapies to patients with rare diseases. The tools and resources for clinical development, particularly large-scale platform cGMP manufacturing and regulatory evaluation of AAV therapies will provide a much-needed standardized approach, enabling meaningful progress for rare disease gene therapy development,” said Christopher Shilling, Senior Vice President of Regulatory Affairs and Quality. “Forge is committed to supporting this collaboration with the NIH, the FDA, and like-minded partners on the BGTC, to provide a critical development platform that will enable the future of AAV therapies for patients.” 

“As one of the largest AAV manufacturers in the world, Forge Biologics has the necessary industry expertise and manufacturing capacity to be a meaningful partner to the BGTC,” said Courtney Silverthorn, Ph.D., Associate Vice President of Science Partnerships at the FNIH, and Program Lead for BGTC. “We are certain their knowledge and capabilities will help the BGTC realize its goal of streamlining the drug development process to reduce costs and enable companies to bring more gene therapies to patients.”  

About the Bespoke Gene Therapy Consortium  
The Bespoke Gene Therapy Consortium (BGTC) is part of the Accelerating Medicines Partnership® (AMP®) program, a public–private partnership among the NIH, the U.S. Food and Drug Administration (FDA), multiple pharmaceutical and life sciences companies, and nonprofit and other organizations. The AMP program, which is managed by the FNIH, aims to improve current models for developing diagnostics and therapies. The BGTC is establishing platforms and standards to speed the development and delivery of customized or “bespoke” gene therapies that could treat millions of people affected by rare diseases, including diseases too rare to be of commercial interest. The BGTC is the first AMP initiative focused on rare diseases and the sixth AMP initiative overall. It also is the first to focus on a therapeutic platform. To learn more, visit https://fnih.org/our-programs/AMP/BGTC.   

About the Foundation for the National Institutes for Health 
The Foundation for the National Institutes of Health (FNIH) connects the world’s leading public and private organizations to accelerate biomedical breakthroughs for patients, regardless of who they are, where they live, or what disease they have. Together with leading scientists and problem-solvers, and a successful track record of navigating complex problems, the FNIH accelerates new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and trains the next generation of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org. 

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life-changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit www.forgebiologics.com.

Media Inquiries
Marina Corleto – Forge Biologics
Associate Director, Marketing and Communications
media@forgebiologics.com   

• Forge fulfills European Medicines Agency’s (EMA) GMP manufacturing standards as determined by a QP audit and declaration to support clients intending to export gene therapy products for clinical programs in Europe

COLUMBUS, Ohio – April 24, 2023 – Forge Biologics, a leading manufacturer of genetic medicines, today announced that its manufacturing facility has successfully completed the audits necessary and received QP declaration for its gene therapy manufacturing facility, thereby fulfilling the European Union Good Manufacturing Practices (EU GMP) requirement to manufacture investigational medicinal products and allowing Forge to support European-based clinical trials.

“QP declaration enhances Forge’s position as a leading global AAV manufacturer able to support clients with the production of high quality therapeutic products destined for European distribution, and helping them to reach patients needing treatments as quickly as possible,” said Christopher Shilling, Senior Vice President of Regulatory Affairs and Quality at Forge.  

A European QP has completed a comprehensive and in-depth audit focused on the quality management systems (QMS) and platform AAV manufacturing process at Forge’s manufacturing facility in Columbus, Ohio, and determined that its facilities, raw materials, suppliers, procedures, and quality systems meet EU GMP requirements. In the EU, a QP is responsible for certifying that each batch of a medicinal product meets all required provisions when released from a manufacturing facility within the EU or imported into the EU. 

Having fulfilled Europe’s GMP requirements, Forge has reached another key regulatory milestone, expanding its CDMO services to support clients in the United States and Europe. Forge clients can rely on the Company’s QP audit as one less hurdle they need to overcome when their AAV gene therapy products are being manufactured for clinical trials and on the path to commercialization in European countries. The QP declaration was received in support of Forge’s internal FBX-101 clinical gene therapy program for the treatment of patients with Krabbe disease.

“The declaration is a significant milestone that demonstrates Forge’s capability to pass the EU QP audit and obtain QP declaration, which will enable us to manufacture products for gene therapy programs to treat patients in the U.S. and Europe,” said Meghan Leonard, Vice President of Quality Management at Forge.  

The QP declaration adds to Forge’s capabilities and makes the Company among one of the few U.S. AAV CDMO’s able to support clients intending to export gene therapy products for clinical programs in Europe.

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life-changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility, the Hearth, utilizes 20 cGMP suites in Columbus, Ohio, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit www.forgebiologics.com.

Media Inquiries
Marina Corleto – Forge Biologics
Associate Director, Marketing and Communications
media@forgebiologics.com