• Manufacturing Partnership to Propel Ray Therapeutics’ Lead Optogenetics Gene Therapy to Phase 1-2 Clinical Trials

SAN DIEGO and COLUMBUS, Ohio – March 1, 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 a manufacturing partnership that will advance Ray Therapeutics’ lead optogenetics gene therapy program, Ray-001, into clinical trials for patients with retinitis pigmentosa.

Forge will provide adeno-associated viral (AAV) process development, scale-up engineering and cGMP manufacturing services for Ray Therapeutics’ program, Ray-001. 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 ft² gene therapy cGMP production facility in Columbus, Ohio.

“We have set up Forge to provide end-to-end gene therapy manufacturing services to enable support for clients striving to make significant improvements in patients’ lives,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge. “We look forward to helping Ray Therapeutics advance their AAV manufacturing with an eye towards restoring vision in patients with ophthalmology disorders.”

Ray-001 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 Ray-001 uses intravitreal administration, from which the vector diffuses into the retina and transduces primarily the RGCs.

“Ray-001 is a ground-breaking new approach to treat inherited retinal diseases using the power of optogenetics, and working together with Forge, we are one step closer to restoring vision in patients who are going blind,” said Paul Bresge, CEO of Ray Therapeutics.

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 Ray-001 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 Diego, 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 has a 200,000+ 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
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, M.S. – Forge Biologics
Chief Commercial Officer
CD@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