- Building on AAV manufacturing partnership with new plasmid production, Forge’s end-to-end manufacturing services will help to advance Ray Therapeutics’ gene therapy for patients living with retinitis pigmentosa and other blinding diseases
SAN FRANCISCO and COLUMBUS, Ohio – October 10, 2022 – Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced further collaboration for their manufacturing partnership to include clinical stage plasmid DNA production to support Ray Therapeutics’ lead optogenetics gene therapy program, RTx-015, in clinical trials for patients with retinitis pigmentosa.
Forge will provide research-grade and GMP-Pathway plasmid manufacturing services, in addition to adeno-associated viral vector (AAV) process development, scale-up engineering, and cGMP manufacturing services for Ray Therapeutics’ program, RTx-015. The program will continue to utilize Forge’s platform manufacturing processes, including its proprietary HEK 293 suspension Ignition Cells™ and pEMBR™ adenovirus helper plasmid. All development and cGMP manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy cGMP production facility in Columbus, Ohio.
“With the recent launch of research and clinical stage plasmid DNA manufacturing, we can better streamline the production of our clients’ AAV programs by vertically integrating plasmids into our manufacturing process,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge. “Our partnership with Ray Therapeutics showcases how these new offerings can accelerate AAV gene therapies from idea into reality for patients in need, and we are excited to help Ray Therapeutics meet the needs of patients with retinitis pigmentosa.”
RTx-015 is intended for use in treating patients with retinitis pigmentosa (RP). Optogenetics is a promising approach that has the potential to restore useful vision to visually-impaired and blind individuals. Patients with RP have damaged photoreceptors, the primary cells required for vision, which are lost and cannot regenerate. However, inner retinal neurons downstream to photoreceptors, especially retinal ganglion cells (RGCs), persist in significant numbers through late-stage disease. Ray Therapeutics’ lead candidate RTx-015 uses intravitreal administration, from which the vector diffuses into the retina and transduces primarily the RGCs.
“By adding clinical grade plasmid production to their existing suite of AAV manufacturing capabilities, Forge is easing the scope of production and accelerating the development of our lead therapeutic,” said Paul Bresge, CEO of Ray Therapeutics. “Offering everything we need under one roof integrates our entire process so that we can focus on our mission to restore vision in patients losing their sight as fast as possible.”
About Retinitis Pigmentosa
Retinitis pigmentosa (RP), is a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. The severe loss of photoreceptor cells that occurs in this genetic degenerative disease leads to partial or complete blindness. At present, no effective treatment is available to restore vision once the photoreceptor cells have been lost. Over 100 genetic mutations are known to cause RP and all types of inheritance patterns are recognized. Patients are typically diagnosed in their late teens, with symptoms including night blindness, reduced visual fields and eventual loss of visual acuity. As the disease progresses, retinal atrophy, and permanent loss of the light sensitive photoreceptors occur. The prevalence of RP is approximately 100,000 persons affected in the US.
About Ray Therapeutics
Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. The company is developing its lead candidate RTx-015 in retinitis pigmentosa, a degenerative retinal disease with significant unmet medical need. The company’s mission is to use optogenetics to restore vision, independent of genetic mutation for patients with inherited retinal diseases. Ray Therapeutics is based in San Francisco, CA. For additional information, please visit www.raytherapeutics.com.
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 focused on AAV manufacturing and can host end-to-end manufacturing services 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
Kimberly Ha – Ray Therapeutics
KKH Advisors
kimberly.ha@kkhadvisors.com
Marina Corleto – Forge Biologics
Senior Manager, Marketing and Communications
media@forgebiologics.com
Client Development
John Maslowski – Forge Biologics
Chief Commercial Officer
CD@forgebiologics.com
- Plasmid and AAV Manufacturing Partnership to Advance Myrtelle’s Novel Gene Therapy for Monogenic Hearing Loss for Phase 1/2 Clinical Trials
WAKEFIELD, Mass., and COLUMBUS, Ohio – October 3, 2022 – Myrtelle Inc., (Myrtelle), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Myrtelle’s novel gene therapy for monogenic hearing loss, Myr-201, into clinical trials for patients with autosomal recessive deafness 8 (DFNB8).
Forge will provide research-grade and GMP-Pathway plasmid manufacturing services as well as cGMP adeno-associated viral (AAV) process development and scale-up manufacturing services for Myrtelle’s program, Myr-201. The program will utilize Forge’s platform process including its proprietary HEK 293 suspension Ignition Cells™ and pEMBR™ adenovirus helper plasmid. All development and cGMP manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy cGMP facility in Columbus, Ohio.
“We are thrilled to be collaborating with Myrtelle to help them accelerate this novel gene therapy for monogenic hearing loss to patients,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics. “This collaboration showcases the full end-to-end capabilities Forge is providing to gene therapy clients. We believe that employing our plasmid manufacturing, coupled with our suite of cGMP AAV manufacturing capabilities and expertise, will help support Myrtelle’s gene therapy technology and skills, enabling this novel therapy to reach those who need it the most.”
Myrtelle entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. (RHI), a private, preclinical stage gene therapy company focusing on diseases affecting human hearing to develop a novel gene therapy for DFNB8 genetic hearing loss that includes low-dose recombinant adeno-associated virus (rAAV) gene therapy delivery of a therapeutic TMPRSS3 (transmembrane protease, serine 3) gene by local administration directly to the inner ear. Across its gene therapy programs, Myrtelle utilizes direct administration of low-dose gene therapy to target key cell types involved in the disorder, thereby avoiding immune-related and off-target effects that can arise with high-dose gene therapy administration delivered systemically. This strategy, currently being developed for Myrtelle’s central nervous system (CNS) programs, can be leveraged to other therapeutic areas outside the CNS, including adjacent and related areas such as the ear where local gene therapy delivery is potentially advantageous for hearing loss disorders such as DFNB8. Preclinical studies in the mouse model of DFNB8-mediated deafness have demonstrated that delivery of a wild type TMPRSS3 gene was able to promote hair cell and neuron survival and improve hearing function.
“This collaboration is an important milestone in Myrtelle’s evolution as we seek to advance our novel gene therapy for monogenic hearing loss which has the potential to become a life-changing genetic medicine,” said Mark Pykett, Chief Executive Officer of Myrtelle. “Partnering with Forge Biologics, a leader in gene therapy manufacturing, and leveraging their AAV expertise, technology, and exceptional facility will support Myrtelle’s development efforts as we move into Phase 1/2 clinical trials with the goal of bringing this transformational therapy to patients in need.”
About DFNB8
Individuals with mutations in TMPRSS3 present with two phenotypes: DFNB10-associated hearing impairment that is pre-lingual and DFNB8-associated hearing impairment that is typically late-onset and post-lingual. TMPRSS3 mutations can be divided into mild or severe; the combination of two severe mutations causes profound pre-lingual hearing loss, whereas milder mutations lead to less severe post-lingual hearing loss.
About Myrtelle
Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company’s website at: www.myrtellegtx.com.
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 focused on AAV manufacturing and can host end-to-end manufacturing services 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
Jordana Holovach – Myrtelle
Head of Communications and Community
jholovach@myrtellegtx.com
Marina Corleto – Forge Biologics
Senior Manager, Marketing and Communications
media@forgebiologics.com
- Enables gene therapy clients to accelerate AAV manufacturing with seamless incorporation into the Company’s HEK 293 platform suspension process with access to end-to-end capabilities
- New offerings of Research-Grade and GMP-Pathway expedite Phase 1/2 clinical trial timelines, with GMP-Grade available in 2023
- Data to support Company’s plasmid production is being presented at BioProcess International East this week
COLUMBUS, Ohio – September 27, 2022 – Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced the new availability of plasmid DNA manufacturing to its suite of scalable manufacturing services for gene therapy programs, complementing Forge’s existing AAV process development, analytical development, cGMP manufacturing, and automated final fill capabilities.
Forge’s plasmid manufacturing services encompass three grades to accommodate a phase-appropriate approach: Research-Grade, suitable for discovery, research and development; GMP-Pathway, suitable for early-stage clinical trial cGMP AAV manufacturing, and cGMP, suitable for late-stage clinical and commercial cGMP manufacturing. Forge’s plasmid production services utilize single-use systems that seamlessly integrate into the Company’s platform AAV manufacturing process, allowing continuity of manufacturing for clients and streamlined vendor management. Research-Grade and GMP-Pathway plasmid grades are currently available, with cGMP plasmid production available in 2023. All plasmid production will occur at Forge’s manufacturing facility, the Hearth, in Columbus, OH, and is available exclusively to clients performing their AAV manufacturing at Forge.
“The launch of plasmid DNA manufacturing enables clients to better streamline the production of their AAV programs by vertically integrating plasmids into our manufacturing process,” said John Maslowski, Chief Commercial Officer of Forge. “The ability to streamline vendor management, better control timeline risks, and accelerate end-to-end AAV manufacturing are important differentiators when upscaling to our 5,000L cGMP bioreactors for clients and patients. We are excited to present the data supporting our now-available services at BioProcess International East this week.”
The Company will deliver data presentations highlighting plasmid production in up to 50L fermenters and AAV manufacturing up to 1,000L at the BioProcess International Conference & Exhibition, taking place in Boston, MA, September 27-30, 2022.
Poster Presentations
Title: Process Development and Scale-Up of Plasmid DNA Production in 1-50L Single Use Fermenters for Accelerating AAV Manufacturing
Presenter: Frank Agbogbo, Ph.D., Vice President, Process Development
Title: Development of a Platform Process for AAV Production
Presenter: Frank Agbogbo, Ph.D., Vice President, Process Development
Title: Development of a Scalable Purification Process for High Grade Plasmid DNA
Presenter: Donald Belcher, Ph.D., Senior Scientist, Process Development
Title: Purification of AAV from 1L to 1,000L Batch Sizes
Presenter: Ganesh Krishnamoorthy, Ph.D., Associate Director, Process Development
Title: Production and the Scale-Up of rAAV from 1L to 1,000L in Bioreactors
Presenter: Steven Wesel, Associate Director, Process Development
Posters will be displayed—and participants can also access them—on the BPI conference website.
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 focused on AAV manufacturing and can host end-to-end manufacturing services 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
Senior Manager, Marketing and Communications
media@forgebiologics.com
Client Development
John Maslowski – Forge Biologics
Chief Commercial Officer
CD@forgebiologics.com