Dystonia Musculorum Deformans

— Company ends 2023 with approximately $123.6 million in cash and investments. Combined with gross proceeds from $108.9 million private placement in January, Solid has anticipated cash runway into 2026 — — FDA cleared IND and granted Fast Track Designation and Orphan Drug Designation for Duchenne muscular dystrophy (Duchenne) gene therapy candidate SGT-003 with patient dosing in Phase 1/2 trial expected Q2 2024 — — Company entered into non-exclusive licensing agreement for use of its proprietary, muscle-targeted AAV-SLB101 capsid — CHARLESTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update. "2023 was an exciting and transformational year for Solid where we completed the integration with AavantiBio, while diversifying and expanding our pipeline. We strengthened our management team with the hiring of a CMO, Dr. Gabriel Brooks, and CFO, Kevin Tan, and advanced our next generation gene therapy for Duchenne, SGT-003, towards the clinic with IND clearance in Q4 and received Fast Track Designation and Orphan Drug Designation in Q4 2023 and Q1 2024, respectively. We continued to bring in additional assets, including SGT-501 for the treatment of a fatal childhood disease called CPVT from ICS Maugeri, while moving additional high potential programs and capsids through preclinical models,” said Bo Cumbo, President and CEO of Solid Biosciences. “We enter 2024 with significant momentum from our recently completed financing in January which raised $108.9 million from a syndicate of leading investors. 2024 will be a year of execution and we look forward to rapidly bringing SGT-003 into the clinic, advancing SGT-501 towards IND filing and most importantly, continuing to bring hope to all those suffering from devastating genetic diseases.” Company Updates Solid expects to initiate dosing in the Phase 1/2 trial of SGT-003 in pediatric patients with Duchenne in the second quarter of 2024. A safety update is expected mid-year and initial data from the first two cohorts of the trial is expected in Q4 2024. Solid announced it expects to IND for its first cardiac gene therapy candidate, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), in the first quarter of 2025. Solid has entered into a non-exclusive licensing agreement for use of its proprietary, muscle-targeted AAV-SLB101 capsid. Solid aims to license AAV-SLB101 broadly to both companies and academic institutions pursuing treatments for rare diseases. Recent Company Highlights On January 16, 2024, Solid announced that the FDA granted Orphan Drug Designation for its Duchenne gene therapy candidate SGT-003. This designation provides certain benefits, including specified financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval. On January 8, 2024, Solid announced a $108.9 million private placement with new and existing investors. The Company expects to use net proceeds of $104.0 million from the private placement to fund ongoing pipeline development programs, business development activities, and for working capital and other general corporate purposes. On December 7, 2023, Solid announced the FDA granted Fast Track Designation for SGT-003. The Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. On November 14, 2023, Solid announced that it had received FDA clearance for the Phase 1/2 trial of SGT-003. Fourth Quarter and Full-Year 2023 Financial Highlights There were no collaboration revenues for the fourth quarter of 2023 and 2022. There were no collaboration revenues for the full year ended December 31, 2023, compared to $8.1 million for the full year ended December 31, 2022. Collaboration revenue in 2022 was related to research services and cost reimbursement from the Collaboration Agreement with Ultragenyx, which the Company entered in the fourth quarter of 2020. Research and development expenses for the fourth quarter of 2023 were $15.4 million, compared to $21.3 million for the fourth quarter of 2022. Research and development expenses for the full year ended December 31, 2023, were $76.6 million, compared to $78.4 million for the full year ended December 31, 2022. The decrease of $1.8 million in research and development expenses was primarily due to a decrease in expenses related to SGT-001 due to our decision to prioritize SGT-003, offset by an increase in costs for SGT-003 and SGT-501. General and administrative expenses for the fourth quarter of 2023 were $6.8 million, compared to $7.6 million for the fourth quarter of 2022. General and administrative expenses for the full year ended December 31, 2023, were $27.8 million, compared to $28.9 million for the full year ended December 31, 2022. Net loss for the fourth quarter of 2023 was $20.3 million, compared to $15.2 million for the fourth quarter of 2022. Net loss for the full year ended December 31, 2023, was $96.0 million, compared to $86.0 million for the full year ended December 31, 2022. The increase in net loss was primarily related to a gain on acquisition and collaboration revenue in 2022, offset by decreased research and development costs, decreased general and administrative expenses, and an increase in yields on cash equivalents and available-for-sale securities in 2023. Solid had $123.6 million in cash, cash equivalents, and available-for-sale securities as of December 31, 2023, compared to $213.7 million as of December 31, 2022. The Company expects that its cash, cash equivalents, and available-for-sale securities as of December 31, 2023, together with the net proceeds from the January 2024 private placement, will enable it to fund key strategic priorities into 2026. About Solid Biosciences Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit . 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 future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company’s priorities and achieve key clinical milestones; the company’s SGT-003 program, including expectations for initiating dosing and availability of clinical trial data; the company’s expectations for submission of an IND for SGT-501; Solid’s plans to license AAV-SLB101 broadly to both companies and academic institutions; the anticipated use of proceeds from the January 2024 private placement; the cash runway of the company and the sufficiency of the Company’s cash, cash equivalents, and available for sale securities 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 the ability to recognize the anticipated benefits of Solid’s acquisition of AavantiBio; the company’s ability to advance SGT-003, SGT-501, AVB-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-501, AVB-401 and other 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 the company’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 the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. Solid Biosciences Contact: Leah Monteiro VP, Investor Relations and Communications 617-766-3430 lmonteiro@solidbio.com

Marsh McLennan Agency's Commitment to Corporate Responsibility Transforms Families' Lives Inside and Outside the Office ATLANTA, Sept. 27, 2023 /PRNewswire/ -- The 2023 Marsh McLennan Agency (MMA) Charity Classic, benefiting the Pediatric Brain Tumor Foundation, will take place on Wednesday, September 27, with an auction and live broadcast, followed by the annual Cocktails & Cornhole event on October 24. Over the past 19 years, employees from Marsh McLennan Agency's Southeast region have impacted the lives of thousands of families in need. Every year, they pour countless hours into working with the Pediatric Brain Tumor Foundation to provide financial relief to families facing the devastating news of a child's brain cancer diagnosis. Pediatric brain cancer is the United States' most commonly diagnosed childhood cancer and deadliest childhood disease, and this health crisis is only growing. More children under the age of 19 are now diagnosed and dying from brain cancer than ever before, while adult brain cancer patients are experiencing declines in mortality rates and incidence of diagnosis. Yet the challenges and barriers families face often go unseen. Federal funding for pediatric brain cancer research and legislation to improve families' access to care aren't keeping up with the growing need, and many states don't include guidance for the needs of childhood cancer patients in their CDC-funded cancer resource plans. Because of the lack of awareness and resources, the economic burden of a pediatric brain tumor diagnosis can be catastrophic. One in four caregivers of a child with cancer lose more than 40% of their median annual income because of work disruptions related to treatment.* Marsh McLennan Agency started their Charity Classic in 2004 to ease the distress so many families face. Their financial support offers a lifeline - providing parents and caregivers a moment of relief so they can focus on the care of their child, not the bill that's due in two days. All funds raised will go towards the Pediatric Brain Tumor Foundation's Butterfly Fund, which provides emergency financial assistance to families of children in treatment at partnering medical facilities. Through the creativity and commitment of their employees, Marsh McLennan Agency's Charity Classic has grown from a golf tournament that raised funds for a handful of families at one hospital into the Pediatric Brain Tumor Foundation's single largest fundraiser: a multi-day auction, live broadcast and cornhole tournament that raises funds for families in 21 hospitals across the country. Marsh McLennan Agency is not only making a larger impact in families' lives than ever before; their support of the Pediatric Brain Tumor Foundation's mission has become a cornerstone of their corporate culture and their engagement with employees on a personal level. Outside of the event, individuals and teams from across the organization volunteer with the Pediatric Brain Tumor Foundation throughout the year. Recently, employees across 11 offices organized a gift card drive to help families pay for gas to their child's treatment and put food on their tables. "Marsh McLennan Agency's dedication to our community transcends what you think of when you think of 'corporate responsibility' with incredible compassion and determination," says Courtney Davies, President and CEO of the Pediatric Brain Tumor Foundation. "Even though most of MMA's employees have not been personally impacted by childhood brain cancer, they take personal responsibility for the hours they dedicate to our mission and show an unwavering commitment to children and families they've never met. We are incredibly grateful for their partnership." For more information about the Marsh McLennan Agency Charity Classic program and the Pediatric Brain Tumor Foundation, visit mma-pbtfcharityclassic.com and curethekids.org. About the Pediatric Brain Tumor Foundation Since 1991, the Pediatric Brain Tumor Foundation's research funding, advocacy, and family support have led the way in ending the childhood cancer community's biggest crisis. Dedicated wholly to addressing this rare, but devastating disease and guided by the experiences of patients, survivors, their parents, and siblings, the Pediatric Brain Tumor Foundation is the only organization to meet families' needs along every step of their cancer journey. The largest patient advocacy funder of pediatric brain tumor research, the foundation also funds and advocates for innovative projects that lead to vital discoveries, new clinical trials, and better treatments – all bringing us closer to a cure. We're able to do that because of people and partners committed to supporting families and ending childhood brain cancer. Learn more at . About Marsh McLennan Agency Marsh McLennan Agency provides business insurance, employee health & benefits, retirement, and private client insurance solutions to organizations and individuals seeking limitless possibilities. With 10,000 colleagues and 170 offices across North America, Marsh McLennan Agency combines the personalized service model of a local consultant with the global resources of the world's leading professional services firm, Marsh McLennan. * Source: Disparities in household material hardship, financial toxicity, and income loss in pediatric cancer, July 2023 SOURCE Pediatric Brain Tumor Foundation

SEATTLE--(BUSINESS WIRE)-- Curi Bio, the industry leader in developing human stem cell-based platforms for drug discovery, proudly announces two recent SBIR grants worth $4.4 million from the National Institutes of Health (NIH) to support its pioneering work on implementing human models to advance discovery of the next generation of medicines. The first project will support expansion of Curi’s cutting-edge in vitro human muscle platform by developing mouse model engineered muscle tissues (EMTs) to bridge the inconsistencies between animal and human data. The second initiative will deliver a novel 3D neuromuscular junction used for disease modeling and potency assays for pharmaceuticals and cosmetics. These grants, the first of their kind received by Curi Bio from the National Center for Advancing Translational Sciences (NCATS), demonstrate the impact that the company's innovative 3D tissue platforms have made in developing new medicines. These latest awards bring the total non-dilutive funding that Curi Bio has received from the NIH to over $12 million. This press release features multimedia. View the full release here: Curi Bio Awarded Over $4.4M in NIH Grants to Advance Translational Science – Read the Press Release for More Details (Graphic: Business Wire) Curi Bio's outstanding success in raising non-dilutive funding via its scientific and technical innovation has been recognized by the U.S. Small Business Administration (SBA), which selected the company as the winner of the 2020 Tibbetts Award. The Tibbetts Award is given to outstanding entities for successfully driving high-tech innovation in the U.S. through the SBA’s Small Business Innovation Research (SBIR) and Small Business Technology Transfer programs. Past awardees include 23andMe, Qualcomm, Symantec, and Broadcom. The company’s first award is focused on developing new in vitro models to address Duchenne muscular dystrophy (DMD), a devastating childhood disease with no known cure and with only a handful of available treatments. During the over $2.3M, two-and-a-half-year project, the company will refine its first-and-only 3D DMD model for use in potency assays, which have been a much-needed tool to address manufacturing bottlenecks and clinical holds amongst the many organizations pursuing novel therapies for the disease. The team will also develop unique 3D engineered heart tissue models based on an industry standard DMD mouse model, enabling drug developers to correlate their decades of mouse data to Curi Bio’s next generation human in vitro models, moving research towards the 3Rs principle of reducing, refining, and replacing animals for preclinical studies. The award is the next advancement from a long-standing collaboration with two investigators at the University of Washington: Dr. Jeffrey S. Chamberlain, a pioneer in DMD therapies whose previous work is already in the clinic, and Dr. David L. Mack, a global authority on neuromuscular diseases and human stem cell and whole-animal models. The second project, awarded by NCATS in conjunction with the Food and Drug Administration as a milestone-based project, is aimed at the pressing need for animal-free potency assays in drug manufacturing. In addition to its many cosmetic uses, Botulinum neurotoxin, commonly known as Botox, is used to treat muscle spasms, chronic pain, and other therapeutic applications. With Botox being the most potent toxin known to man, safe and effective dosing relies on accurate methods to assess the potency of manufactured lots. Current practices utilize the mouse lethality bioassay, which is at odds with regulatory strategies striving to implement the 3Rs. Curi Bio will develop a novel neuromuscular junction model from human stem cells that can measure the effect of the toxin based on a high-throughput assay that can be readily adapted to study myriad neuromuscular disorders. This work will be performed in conjunction with the laboratory of Dr. Alec Smith and Dr. David Mack at the University of Washington, Seattle. "These awards from the NIH represent a major validation of the importance of our innovative platforms in advancing pre-clinical research and developing sorely needed potency assays for the next generation of medicines," said Dr. Nicholas Geisse, CEO of Curi Bio and Principal Investigator on both awards. "We are honored to receive this recognition from NCATS and are committed to leveraging this funding to drive our internal R&D efforts forward, furthering our mission of accelerating the safe and effective development of new therapeutics." The prestigious Fast-Track grants, which amount to a total more than $4.4 million over a two-and-a-half-year period, will provide Curi Bio with the necessary resources to propel its groundbreaking research in DMD as well as development of a Botox potency screening assay using a novel neuromuscular junction model. With this substantial funding, Curi Bio aims to accelerate the commercialization of its next generation 3D EMT platforms as powerful research tools for drug developers and biopharmaceutical companies. Research reported in this press release is supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Numbers R44TR004557 and U44TR004795. This content is solely the responsibility of Curi Bio and does not necessarily represent the official views of the National Institutes of Health. About Curi Bio Curi Bio’s preclinical discovery platform combines human stem cells, systems, and data to accelerate the discovery of new medicines. The Curi Engine™ is a comprehensive, bioengineered platform that integrates human iPSC-derived cell models, tissue-specific biosystems, and AI/ML-enabled phenotypic screening data. Curi Bio’s suite of human stem cell-based products and services enable scientists to build more mature and predictive human iPSC-derived tissues—with a focus on cardiac, musculoskeletal, and neuromuscular models—for the discovery, safety testing, and efficacy testing of new drugs in development. By offering drug developers an integrated preclinical platform comprising highly predictive human stem cell models to generate clinically-relevant data, Curi Bio is closing the gap between preclinical data and human results, accelerating the discovery and development of safer, more effective medicines. For more information, please visit . View source version on businesswire.com: Contacts For media inquiries, please contact: Heejoon Choi Director of Sales & Marketing, Curi Bio heejoon@curibio.com Source: Curi Bio Smart Multimedia Gallery Photo Curi Bio Awarded Over $4.4M in NIH Grants to Advance Translational Science – Read the Press Release for More Details (Graphic: Business Wire) Logo View this news release and multimedia online at: