• 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   

• Partnership to Bolster Solid Biosciences’ Gene Therapy Pipeline, Including AAV Process Development, Scale Up and cGMP Manufacturing Services

CAMBRIDGE, Mass., and COLUMBUS, Ohio – October 4, 2021 – Solid Biosciences Inc. (Solid, Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), and Forge Biologics, a cell and gene therapy-focused contract development and manufacturing organization (CDMO), announced a partnership to advance the development and manufacturing of SGT-003, Solid’s next generation gene therapy program for Duchenne. SGT-003 is a preclinical candidate that combines a next-generation and rationally designed capsid with Solid’s proprietary nNOS-containing microdystrophin and has demonstrated enhanced muscle tropism and microdystrophin expression compared to AAV9 in vivo.

Forge will provide an adeno-associated viral (AAV) vector process, scale-up engineering and cGMP manufacturing services for SGT-003. The program will employ Forge’s Blaze™ Vector production platform, and Forge’s proprietary HEK293 suspension Ignition™ Cells and pEMBR™ adenovirus helper plasmid, to support Solid’s clinical development. All development and cGMP manufacturing activities will occur at The Hearth, Forge’s 175,000 ft² gene therapy cGMP production facility in Columbus, Ohio.

“We are excited to partner with Forge, a company who shares our high standards for product purity, potency and reproducibility, to further our ability to bring meaningful therapies to patients with Duchenne,” said Joel Schneider, Ph.D., Chief Operating Officer of Solid Biosciences. “As we continue to develop our pipeline, it is important that we have partners who will enhance our expertise. Uniting Forge’s integrated platforms and cGMP gene therapy manufacturing capabilities with our in-depth knowledge in high dose gene therapy development and manufacturing will introduce an additional method to produce AAV gene therapy at Solid and help to accelerate human proof of concept for SGT-003.”

“We are thrilled to partner with Solid and look forward to providing support and cGMP manufacturing services as they advance their next-generation AAV gene therapy for Duchenne,” said Timothy J. Miller, Ph.D., Chief Executive Officer, President, and Co-Founder of Forge. “Forge’s flexible and scalable manufacturing offerings are an ideal complement to Solid’s development efforts with the shared goal of advancing potential treatments for patients with Duchenne.”

Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the ability of  Solid to continue dosing patients in the IGNITE DMD trial, the implication of interim clinical data, the safety or potential treatment benefits of SGT-001 in patients with DMD, Solid’s regulatory plans, the Company’s SGT-003 program, including Solid’s expectation for filing an IND, timelines, the sufficiency of Solid’s cash and cash equivalents to fund its operations, and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Solid’s ability to continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll additional patients in IGNITE DMD and on the timeline expected; Solid’s dosing strategy; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; whether the interim data referenced in this release will be predicative of the final results of the trial or will demonstrate a safe or effective treatment benefit of SGT-001; whether the methodologies, assumptions and applications Solid utilizes to assess particular safety or efficacy parameters will yield meaningful statistical results; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, SGT-003 and other product candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Solid’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in Solid’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Solid’s views as of the date hereof and should not be relied upon as representing Solid’s views as of any date subsequent to the date hereof. Solid anticipates that subsequent events and developments will cause Solid’s views to change. However, while Solid may elect to update these forward-looking statements at some point in the future, Solid specifically disclaims any obligation to do so.

About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive—work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001. For more information, please visit www.solidbio.com.

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge has a 175,000 square foot facility in Columbus, Ohio, The Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will 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
Caitlin Lowie – Solid Biosciences
VP, Communications and Investor Relations
clowie@solidbio.com

Marina Corleto – Forge Biologics
Senior Manager, Marketing and Communications
media@forgebiologics.com   

Families and Clinician Inquiries
Dr. Maria Escolar – Forge Biologics
Chief Medical Officer
medicalaffairs@forgebiologics.com

Business Development  
Magdalena Tyrpien – Forge Biologics
Vice President, Head of Business Development  
BD@forgebiologics.com   

Client Development
John Maslowski – Forge Biologics
Chief Commercial Officer
CD@forgebiologics.com

Investor Relations  
Christina Perry, MSA, CPA – Forge Biologics
Vice President, Finance and Investor Relations  
Investors@forgebiologics.com   

• Partnership to support GentiBio’s cell therapy pipeline with AAV process development, scale up and GMP manufacturing services

BOSTON, SEATTLE, and COLUMBUS, Ohio – July 15, 2021 – GentiBio, Inc., an emerging biotherapeutics company developing best-in-class engineered regulatory T cells programmed to treat autoimmune, alloimmune, autoinflammatory, and allergic diseases, and Forge Biologics, a cell and gene therapy-focused contract development and manufacturing organization (CDMO), announced a strategic development and manufacturing partnership today to advance GentiBio’s unique immune tolerance platform.

Forge will provide adeno-associated viral (AAV) vector process and analytical development, scale-up engineering and GMP manufacturing services for GentiBio’s engineered Tregs therapeutic candidates. The program will employ both Forge’s Blaze™ Vector production platform and HEK 293 Suspension Ignition™ Cells to facilitate GentiBio’s development process. All development and GMP activities will occur at The Hearth, Forge’s 175,000 ft² gene therapy GMP production facility in Columbus, Ohio.

“We are thrilled to partner with Forge Biologics and their experienced team to further develop our lead engineered Treg program and take a critical step in advancing our therapies to patients in need,” said Andy Walker, Ph.D., co-founder and Chief Technology Officer of GentiBio. “Forge offers a broad array of services ranging from process development to GMP manufacturing, making them a strong partner for GentiBio’s cell therapies with capacity to support GentiBio as we advance programs through clinical development.”

Forge’s Hearth is the home of a state-of-the-art cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate cell and gene therapy programs from preclinical through clinical and commercial stage manufacturing. Forge offers product and analytical development services, cGMP manufacturing and regulatory support services.

“Our team is excited to embark on this partnership with GentiBio to meet their viral vector manufacturing needs and help them advance to clinical trials,” said Timothy J. Miller, Ph.D., Chief Executive Officer, President, and Co-Founder of Forge. “Forge is committed to providing quality AAV manufacturing to all stages of program development, and this partnership highlights the Forge team’s capabilities to help innovators such as the GentiBio team on their path to success.”

About GentiBio
GentiBio, Inc., is an emerging biotherapeutics company co-founded by pioneers in Treg biology and synthetic immunology to develop engineered regulatory T cells programmed to treat autoimmune, alloimmune, autoinflammatory and allergic diseases. GentiBio’s proprietary autologous and allogeneic EngTregs platform integrates key complementary technologies needed to successfully restore immune tolerance and overcome major limitations in existing regulatory T cell therapeutics. GentiBio is at the forefront of leveraging a unique therapeutic modality that can be used to address the fundamental cause of many diseases that result from overactivity and/or malfunctioning of the immune system. To learn more, visit https://www.gentibio.com

About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft² facility in Columbus, Ohio, The Hearth, to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will 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 is building on the theme of “Hope in Ohio” to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit www.forgebiologics.com

Media Inquiries
Marites Coulter – GentiBio
Verge Scientific, Inc.
mcoulter@vergescientific.com

Dan Salvo – Forge Biologics
Director of Communications and Community Development
media@forgebiologics.com

Business Development
Magdalena Tyrpien – Forge Biologics
Vice President and Head of Business Development
BD@forgebiologics.com

Investor Relations
Christina Perry – Forge Biologics
Vice President, Finance and Operations
Investors@forgebiologics.com