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Solid Biosciences Inc. (Nasdaq: SLDB) is a Cambridge, MA-based biotechnology company focused on developing treatments and cures for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease. Solid Biosciences combines commercial acumen with top-tier science to advance promising therapies that address all aspects of DMD. The core of Solid's mission is to improve the daily lives of patients and target the roots of this debilitating condition.
The company's primary focus is on developing gene therapies and other treatments for neuromuscular and cardiac diseases. Their product pipeline includes:
- SGT-003: A next-generation gene therapy candidate for DMD, recently granted FDA clearance for an Investigational New Drug (IND) application. The therapy uses a proprietary capsid (AAV-SLB101) to deliver a microdystrophin transgene to promote muscle function and durability. The planned Phase 1/2 trial aims to evaluate the safety and tolerability of SGT-003 in pediatric patients.
- SGT-501: A treatment for catecholaminergic polymorphic ventricular tachycardia (CPVT), a fatal childhood disease.
- AVB-401: For the treatment of BAG3-mediated dilated cardiomyopathy.
- AVB-202-TT: Aimed at treating Friedreich’s ataxia.
Solid Biosciences has made significant strides in obtaining regulatory milestones. Recently, SGT-003 was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the FDA, enhancing its potential marketability and support for clinical development.
The company has also been involved in strategic collaborations and financing activities. Notably, Solid entered a licensing agreement with Armatus Bio for the use of its AAV-SLB101 capsid, securing a non-exclusive worldwide license and upfront and milestone payments. In January 2024, they raised $108.9 million from a private placement to support ongoing pipeline development and business activities.
Financially, Solid Biosciences reported a net loss of $96.0 million for 2023 but expects its cash reserves and recent financing to fund strategic priorities into 2026. As of December 31, 2023, the company had $123.6 million in cash, cash equivalents, and available-for-sale securities.
For more detailed information, visit www.solidbio.com.
Solid Biosciences Inc. (Nasdaq: SLDB) has granted an inducement award to Dr. Roxana Dreghici, the newly appointed Senior Vice President of Clinical Development. This grant, approved by independent directors on June 16, 2021, includes an option to purchase 230,000 shares of common stock at an exercise price of $3.77 per share, reflecting the closing price on July 1, 2021. The option vests over four years, contingent on Dr. Dreghici's continued employment. Solid Biosciences is focused on developing therapies for Duchenne muscular dystrophy, particularly through its lead gene therapy candidate, SGT-001.
Solid Biosciences (Nasdaq: SLDB) has announced the hiring of three key executives: Roxana Donisa Dreghici as Senior Vice President of Clinical Development, Caitlin Lowie as Vice President of Communications & IR, and Alison Kessler as Vice President of Legal Corporate & IP. These appointments are aimed at strengthening the management team and advancing the company's product pipeline focused on Duchenne muscular dystrophy. Each new executive brings significant industry experience, enhancing Solid Biosciences' capacity to support patients and their families affected by this condition.
Solid Biosciences (Nasdaq: SLDB) has announced the grant of an inducement award to Dr. Iman Barilero, the newly appointed Chief Regulatory Officer. This award consists of an option to purchase 180,000 shares at an exercise price of $3.75 per share, based on the closing stock price as of June 1, 2021. The option vests in four equal installments over four years, contingent upon Dr. Barilero's ongoing service with the company. Solid Biosciences focuses on developing therapies for Duchenne muscular dystrophy, targeting the underlying genetic mutation with its lead candidate, SGT-001.
Solid Biosciences (Nasdaq: SLDB) announced Dr. Iman Barilero as the new Chief Regulatory Officer, effective June 1, 2021. Dr. Barilero brings over 30 years of regulatory experience, having successfully guided numerous drug approvals in various global markets. She previously served as Senior Advisor to Innouvo and held key positions at Agenus, Poxel, and Lundbeck. Her expertise aligns with Solid’s mission to enhance therapies for Duchenne muscular dystrophy, particularly focusing on SGT-001 and SGT-003. The company aims to develop differentiated therapies through innovative regulatory strategies.
Solid Biosciences (Nasdaq: SLDB) announced that its COO, Joel Schneider, PhD, will present at the Jefferies Virtual Healthcare Conference on June 3, 2021, at 3:30 p.m. ET. A live webcast will be accessible on the Company’s investor relations website, with a replay available after the event. Solid Biosciences focuses on developing therapies for Duchenne muscular dystrophy, specifically targeting the underlying mutations with its lead gene therapy candidate, SGT-001.
Solid Biosciences (Nasdaq: SLDB) announced participation in key scientific conferences focusing on Duchenne muscular dystrophy. At the 16th International Congress of Neuromuscular Diseases on May 21-22 & 28-29, Dr. Perry Shieh will present findings from the Phase I/II IGNITE DMD trial of SGT-001 microdystrophin gene therapy, highlighting long-term biopsy data and safety evaluations. Additionally, at the Gene Therapy for Muscular Disorders conference from May 25-27, Dr. Carl Morris will discuss drug development strategies and participate in expert panels.
Solid Biosciences (SLDB) provided an update on its Q1 2021 performance, with a net loss of $16.9 million, down from $26.7 million in Q1 2020. The company reported collaboration revenue of $3.3 million and ended the quarter with $268.5 million in cash, ensuring a cash runway into Q4 2022. In clinical updates, two additional patients were dosed in the IGNITE DMD trial, with a serious adverse event noted for patient 8. Long-term results showed durable microdystrophin expression from SGT-001, and the company announced advancements in its next-generation DMD gene therapy candidate, SGT-003.
Solid Biosciences (Nasdaq: SLDB) announced it will release its first quarter 2021 financial results before the market opens on May 14, 2021. A conference call will be held at 8:30 a.m. ET to discuss the financial results and recent business updates. The event can be accessed via a live webcast on the company's website, with an archived version available afterward. Solid Biosciences focuses on developing therapies for Duchenne muscular dystrophy, primarily through its lead gene therapy candidate, SGT-001.
Solid Biosciences (Nasdaq: SLDB) announced the presentation of three abstracts at the ASGCT 24th Annual Meeting, focusing on AAV vectors and the IGNITE DMD trial for SGT-001, a gene therapy for Duchenne muscular dystrophy. Key presentations include:
- AAV9 immune response mechanisms.
- Characterization of novel AAV vectors for muscle gene delivery.
- 12-month data from the IGNITE DMD Phase I/II trial of SGT-001, scheduled for May 14, 2021.
SGT-001 aims to address the genetic causes of Duchenne by delivering a synthetic dystrophin gene.
Solid Biosciences (Nasdaq: SLDB) announced participation in a virtual fireside chat at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on April 27, 2021, at 10:30 am ET. Joel Schneider, COO, and Louise Perry, Head of Technical Operations, will discuss advancements in therapies for Duchenne muscular dystrophy.
A live webcast will be available on the Company's investor relations website, and a replay can be accessed afterwards.
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