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Sarepta Drops 42% on Fatalities; Markets Eye Solid Biosciences
MarketBeat· 2025-06-18 15:39
Sarepta Therapeutics TodaySRPTSarepta Therapeutics$21.56 +0.00 (+0.02%) 52-Week Range$18.30▼$173.25Price Target$70.76Add to WatchlistIn another tragic incident, Sarepta Therapeutics NASDAQ: SRPT saw a second death in connection with its Duchenne muscular dystrophy (DMD) treatment ELEVIDYS. The patient passed away due to acute liver failure (ALF) after doctors treated them with ELEVIDYS. Sadly, the same thing happened three months ago. As a result, Sarepta has suspended shipments of ELEVIDYS for non-ambulat ...
Solid Biosciences (SLDB) 2025 Conference Transcript
2025-06-04 19:02
Solid Biosciences (SLDB) 2025 Conference June 04, 2025 02:00 PM ET Speaker0 Everyone. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the SolidBio team. We've got Bo Kumbow and Gabe Brooks joining us today. And we're going to do fireside chat format. So, for those who are new to the story, maybe give a brief intro to Solid, your key programs and platform. Speaker1 Yeah, thank you, Mari. I appreciate the invitation. Solid Bioscienc ...
Solid Biosciences to Participate at the Jefferies Global Healthcare Conference
Globenewswire· 2025-05-28 12:00
Solid Biosciences Investor Contact: CHARLESTOWN, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, at 2:00 pm ET. A live webcast of the fireside chat will b ...
Solid Biosciences Reports First Quarter 2025 Financial Results and Provides Business Updates
Globenewswire· 2025-05-15 20:06
- Duchenne (SGT-003): Participant dosing ongoing in the Phase 1/2 INSPIRE DUCHENNE trial; Solid on track to discuss accelerated pathways with U.S. FDA later in 2025 - - FA (SGT-212): Dosing of first participant anticipated in the second half of 2025 - - CPVT (SGT-501): FDA IND on track for submission first half of 2025 - - Cash: Company ended first quarter 2025 with $306.9 million in cash, cash equivalents, investments and available-for-sale securities; Solid has anticipated cash runway into the first half ...
Solid Biosciences(SLDB) - 2025 Q1 - Quarterly Report
2025-05-15 20:04
FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 90-0943402 (Stat ...
Solid Biosciences (SLDB) 2025 Conference Transcript
2025-05-07 17:30
Summary of Solid Biosciences (SLDB) Conference Call Company Overview - **Company**: Solid Biosciences (SLDB) - **Recent Changes**: Solid Biosciences underwent a transformation following a merger with Avanti Bio, focusing on advancing gene therapy technologies, particularly for Duchenne Muscular Dystrophy (DMD) [4][5][6] Core Points and Arguments - **Gene Therapy Focus**: Solid Biosciences emphasizes the importance of delivery mechanisms in gene therapy, which includes capsids, promoters, and manufacturing processes [5][6] - **Next Generation Therapies**: The company is developing next-generation gene therapies that aim to provide better clinical outcomes and true value, with a focus on DMD [6][7] - **Clinical Trials**: Solid is currently conducting clinical trials for SGT003, which is positioned as a leading gene therapy for DMD, featuring advanced capsid and manufacturing processes [6][8] - **Unique Transgene Design**: SGT003 utilizes a novel transgene design that enhances flexibility and durability of the protein, which is crucial for muscle function [9][10] - **Clinical Data**: Initial results from the INSPIRE trial show promising outcomes, including high levels of muscle fiber positivity and significant NOS activity, indicating potential benefits for muscle integrity and cardiac health [11][12][15][17][20][25] Key Metrics and Data - **Muscle Fiber Positivity**: 80% of muscle fibers were positive at the intermediate biopsy, indicating effective transduction [14] - **Dystrophin Sarcoglycan Complex**: 70% of the complex was shown to come together, a significant achievement in DMD research [15] - **NOS Activity**: A 62-fold increase in NOS activity was observed, with 42% of muscle fibers exhibiting NOS activity [15][17] - **Cardiac Health Indicators**: Elevated troponin levels were normalized post-treatment, and improvements in ejection fraction were noted in treated patients [23][25] Market Position and Future Outlook - **Market Potential**: Solid Biosciences is targeting a significant patient population, with approximately 400 new DMD patients born annually in the US and a larger population in Europe and beyond [36][37] - **Regulatory Strategy**: The company plans to meet with the FDA in Q4 to discuss accelerated approval pathways for their therapies [27][32] - **Pipeline Expansion**: Solid is also advancing other gene therapies, including SGT-212 for Friedrich's Ataxia and a drug for catecholaminergic polymorphic ventricular tachycardia (CPVT), indicating a broadening of their therapeutic focus [38][44] Additional Insights - **Response to Market Events**: The company noted an influx of patients wanting to enroll in trials following adverse events in competitor trials, highlighting the competitive landscape and patient demand for effective therapies [28][29] - **Community Engagement**: Solid has actively engaged with the DMD community, indicating strong interest and support for their clinical trials [39][41] - **Unique Selling Proposition**: Solid Biosciences positions itself as a leader in gene therapy by focusing on comprehensive treatment approaches that address both cardiac and muscular aspects of diseases [42][43]
Solid Biosciences to Participate at The Citizens Life Sciences Conference
Globenewswire· 2025-04-30 12:00
CHARLESTOWN, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, will participate in a fireside chat at The Citizens Life Sciences Conference on Wednesday, May 7, at 12:30 pm ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by cli ...
Solid Biosciences to Present at the American Society of Gene and Cell Therapy’s 28th Annual Meeting
Globenewswire· 2025-04-29 12:00
1. Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy Session Title: Late-Breaking Abstracts II Date/Time: May 17, 9:15 – 9:30am CT Location/Room: Room 391-392 Presenter: Kevin Flanigan, MD, Advisor Consultant & Researcher, Nationwide Children's Hospital and investigator in the INSPIRE DUCHENNE clinical trial 2. Title: Insight into the Mechanism of Action of AAV-SLB101, a Novel Muscle-Tropic Capsid for Neuromuscular an ...
Solid Biosciences: A Strong Buy As An Emerging Biotech In The Gene Therapy Space
Seeking Alpha· 2025-04-08 20:50
Group 1 - Solid Biosciences Inc. (NASDAQ: SLDB) is potentially repositioning itself in a sector that has recently faced significant scrutiny [1] - The company's lead candidate is still in early-stage trials but has demonstrated encouraging results [1]
Can Solid Biosciences Challenge Sarepta in the DMD Market?
MarketBeat· 2025-04-08 11:31
When it comes to treating Duchenne muscular dystrophy (DMD), big news recently surfaced. DMD is a muscle disease. It usually first shows up in boys aged two to five years. Genetic alterations cause it, and the condition progressively gets worse over time. Solid Biosciences TodaySLDBSolid Biosciences$2.94 -0.18 (-5.77%) 52-Week Range$2.77▼$12.32Price Target$15.67Add to WatchlistCurrently, the only Food and Drug Administration (FDA)-approved gene therapy to treat DMD is ELEVIDYS. Biotech company Sarepta Ther ...