Solid Biosciences Provides 2026 Outlook Underscoring Neuromuscular and Cardiac Pipeline Momentum and Expanded Access to Next-Generation Capsid AAV-SLB101

Rhea-AI Summary

Solid Biosciences (NASDAQ: SLDB) provided a 2026 outlook highlighting clinical progress across neuromuscular and cardiac programs and expanded access to its next‑generation capsid AAV‑SLB101.

Key points: 33 participants dosed with SGT‑003 in the Phase 1/2 INSPIRE Duchenne trial (safety cutoff 1/9/2026) with outpatient dosing enabled since Sept 2025; first participant enrolled in ex‑U.S. Phase 3 IMPACT Duchenne with dosing expected Q1 2026; first participant dosed in Phase 1b FALCON for SGT‑212 (FA); ARTEMIS sites activated for SGT‑501 (CPVT); FDA granted Orphan, Rare Pediatric Disease and Fast Track for SGT‑212; executed 50+ agreements for AAV‑SLB101.

Positive

• SGT-003: 33 participants dosed as of 1/9/2026
• Outpatient dosing enabled for SGT-003 since September 2025
• SGT-212: first participant dosed in Phase 1b FALCON
• SGT-212 awarded Orphan, Rare Pediatric Disease, Fast Track
• 50+ agreements executed for capsid AAV-SLB101 access

Negative

• Phase 3 IMPACT Duchenne dosing expected Q1 2026 but enrollment-dependent
• Mid-2026 data and FDA alignment contingent on future interactions
• Key biomarker and safety data from small cohorts (N=10–14)
• Ex-U.S. trial expansion subject to regulatory approvals

News Market Reaction

-1.53% 1.5x vol

43 alerts

-1.53% News Effect

+10.0% Peak in 4 hr 59 min

-$7M Valuation Impact

$464M Market Cap

1.5x Rel. Volume

On the day this news was published, SLDB declined 1.53%, reflecting a mild negative market reaction. Argus tracked a peak move of +10.0% during that session. Our momentum scanner triggered 43 alerts that day, indicating elevated trading interest and price volatility. This price movement removed approximately $7M from the company's valuation, bringing the market cap to $464M at that time. Trading volume was above average at 1.5x the daily average, suggesting increased trading activity.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

INSPIRE DUCHENNE participants: 33 participants Cardiac monitoring sample: N=14 Microdystrophin expression sample: N=10 +5 more

8 metrics

INSPIRE DUCHENNE participants 33 participants Dosed in Phase 1/2 INSPIRE DUCHENNE trial as of Jan 9, 2026

Cardiac monitoring sample N=14 LVEF cardiac systolic function data cutoff Sept 29, 2025

Microdystrophin expression sample N=10 Compelling microdystrophin expression data cutoff Sept 29, 2025

AAV-SLB101 agreements 50+ agreements Licenses and access deals for next-generation capsid AAV-SLB101

Net loss $45.8 million Q3 2025 net loss from 10-Q filing

Cash and equivalents $236.1 million Cash, cash equivalents and securities as of Sept 30, 2025

Capital raised $188.0 million Net proceeds from February 2025 offering noted in 10-Q

Funding runway into first half of 2027 Management liquidity guidance from Q3 2025 10-Q

Market Reality Check

Price: $5.47 Vol: Volume 1,298,451 is 35% a...

normal vol

$5.47 Last Close

Volume Volume 1,298,451 is 35% above the 20-day average of 964,543, indicating elevated interest ahead of the update. normal

Technical Shares at $5.24 are trading above the 200-day MA of $4.95, while sitting 28.9% below the 52-week high and above the $2.41 52-week low.

Peers on Argus

SLDB slipped 0.95% with above-average volume, while key biotech peers were mixed...

SLDB slipped 0.95% with above-average volume, while key biotech peers were mixed: FULC fell 8.47%, AURA was modestly lower, and LXRX and ALMS gained 2.61% and 7.88% respectively. The pattern points to stock-specific trading rather than a coordinated sector move.

Historical Context

5 past events · Latest: Jan 12 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 12	Regulatory designation	Positive	-0.9%	FDA Orphan Drug designation and dual-route FA gene therapy update.
Jan 12	Clinical trial update	Positive	-0.9%	First participant dosed in FALCON Phase 1b FA trial SGT-212.
Jan 06	Conference participation	Neutral	+3.0%	Announcement of J.P. Morgan Healthcare Conference presentation webcast.
Jan 05	Equity incentives	Neutral	-2.2%	Inducement grant of 7,000 RSUs under 2024 Inducement Plan.
Dec 16	Policy milestone	Positive	+1.9%	Duchenne added to U.S. RUSP, supporting newborn screening and access.

Pattern Detected

Recent clinically positive and designation-related news has not consistently translated into positive next-day price moves, with several favorable updates followed by declines.

Recent Company History

Over the last month, Solid Biosciences has highlighted growing momentum across its Duchenne and Friedreich’s ataxia programs. On Dec. 16, 2025, the company benefited from Duchenne’s addition to the U.S. RUSP, supporting earlier diagnosis while it advances SGT-003. In early January, SLDB announced a J.P. Morgan presentation (Jan. 13, 2026) and reported inducement equity grants. On Jan. 12, 2026, it disclosed Orphan Drug designation and first dosing in the FALCON trial for SGT-212. Today’s broad 2026 pipeline outlook builds directly on these recent regulatory and clinical milestones.

Market Pulse Summary

This announcement highlights Solid Biosciences’ transition into 2026 with four active clinical trial...

Analysis

This announcement highlights Solid Biosciences’ transition into 2026 with four active clinical trials and expanding use of its AAV-SLB101 capsid, including 33 Duchenne patients dosed in INSPIRE DUCHENNE and the first Phase 3 participant enrolled ex-U.S. The update builds on recent FDA Orphan Drug status and first dosing for SGT-212. Investors may watch upcoming regulatory interactions on an accelerated pathway for SGT-003, initial FALCON and ARTEMIS data in 2026, and the company’s cash trajectory against its R&D commitments.

Key Terms

Phase 3 randomized, double-blind, placebo-controlled, Orphan Drug designation, Fast Track, Rare Pediatric Disease, +4 more

8 terms

Phase 3 randomized, double-blind, placebo-controlled technical

"a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial"

A phase 3 randomized, double-blind, placebo-controlled trial is a large, late-stage clinical study that tests whether a new treatment works and is safe by assigning participants by chance to either the treatment or an inactive pill, with neither patients nor researchers knowing who gets which. It matters to investors because passing this rigorous “final dress rehearsal” is a major step toward regulatory approval, market access, and potential revenue, while failure can sharply reduce a drug’s commercial prospects.

Orphan Drug designation regulatory

"FDA Orphan Drug designation granted to SGT-212"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

Fast Track regulatory

"SGT-212 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track designations"

A fast track designation is a regulatory label that speeds up the review and communication between a drug developer and regulators for treatments addressing serious illnesses or unmet medical needs. For investors, it matters because it can shorten development time and reduce regulatory delays—like getting a VIP lane at the airport—raising the chance of earlier market access and potential revenue, though it does not guarantee approval.

Rare Pediatric Disease regulatory

"Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA"

A rare pediatric disease is a serious medical condition that primarily affects children and occurs so infrequently that only a small number of patients exist. Investors care because treatments for such conditions often get special regulatory incentives—think of government fast lanes and rewards for developers—making smaller markets potentially profitable due to pricing power, shorter development timelines, and reduced competition, much like a niche product that receives government-backed advantages.

left ventricular ejection fraction (LVEF) medical

"normalization, as measured by left ventricular ejection fraction (LVEF) (N=14"

Left ventricular ejection fraction (LVEF) is the percentage of blood the heart’s main pumping chamber pushes out with each beat, a simple measure of how effectively the heart pumps like the efficiency rating on a water pump. Investors care because LVEF is a key clinical indicator used to diagnose and guide treatment for heart failure and other cardiac conditions, which affects demand for drugs, devices, reimbursement, trial outcomes, and company valuation.

thrombotic microangiopathy (TMA) medical

"No drug-induced liver injury (DILI), thrombotic microangiopathy (TMA), atypical"

Thrombotic microangiopathy (TMA) is a medical condition where small blood vessels become blocked by clots and damaged cells, which can quickly impair organ function like the kidneys or brain. For investors, TMA matters because it is a serious safety signal in drug development and patient care—its occurrence can trigger regulatory action, costly trials or label changes, litigation risk, and sudden shifts in a company’s market value, much like a product recall affects a manufacturer.

atypical hemolytic uremic syndrome (aHUS) medical

"thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS) or"

Atypical hemolytic uremic syndrome (aHUS) is a rare, serious condition where the body’s immune system misfires and causes tiny blood clots that destroy red blood cells and block small blood vessels in the kidneys, often leading to sudden kidney failure. It matters to investors because aHUS drives demand for specialized treatments, influences clinical trial and regulatory outcomes, and can affect a company’s revenue, liability and pipeline valuation in the rare-disease market.

catecholaminergic polymorphic ventricular tachycardia (CPVT) medical

"trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT)"

A rare inherited heart rhythm disorder that causes dangerously fast, irregular heartbeats when the body releases stress hormones such as during exercise or emotional upset; it can lead to fainting or sudden cardiac arrest if untreated. Investors care because it represents a specific medical need where diagnostics, drug therapies, or implantable devices can have clear clinical benefit and commercial value, much like fixing faulty wiring that only shorts during high demand.

AI-generated analysis. Not financial advice.

- Duchenne: Dosed 33 participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of January 9, 2026; SGT-003 continues to be generally well tolerated using a steroid-only prophylactic immunomodulation regimen -

- Duchenne: First participant enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected in Q1 2026 -

- FA: First participant dosed in Phase 1b FALCON clinical trial; FDA Orphan Drug designation granted to SGT-212, the only dual route of administration gene therapy in development to treat Friedreich’s ataxia (FA) -

- CPVT: Clinical trial sites activated for ARTEMIS, a Phase 1b first-in-human clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT); participant screening is underway -

- Capsids (AAV-SLB101): Executed 50+ agreements, including licenses, with corporations, institutions and academic labs for the use of proprietary, next-generation capsid AAV-SLB101 -

CHARLESTOWN, Mass., Jan. 13, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will provide a corporate update outlining progress in advancing its neuromuscular and cardiac gene therapy programs in a presentation delivered by Bo Cumbo, President and CEO, at the 44^th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:15 p.m. PT (8:15 p.m. ET).

“Over the past year, we have executed across our pipeline, building critical momentum as we enter 2026 with four active clinical trials in three devastating neuromuscular and cardiac rare diseases with significant unmet need, including the recently announced dosing of the first participant in our Phase 1b FALCON trial evaluating SGT-212 for the treatment of FA,” said Mr. Cumbo. “By rapidly and responsibly advancing innovative science and forging collaborations to enhance gene therapy delivery using our next-generation capsid, we are determined to build a better future for the patient communities we serve while driving value for our shareholders. With multiple regulatory interactions planned in the coming months, we aim to achieve alignment on a potential accelerated approval pathway for SGT-003 and to bring our investigational therapy to market to meet the overwhelming demand from the Duchenne community.”

Highlights from the presentation to be given at the J.P. Morgan Healthcare Conference include:

Neuromuscular Pipeline
SGT-003 for Duchenne muscular dystrophy (Duchenne)

U.S.:

• As of a safety cutoff date of January 9, 2026, SGT-003 has been generally well tolerated in the 33 participants dosed in the ongoing INSPIRE DUCHENNE Phase 1/2 clinical trial.
  • SGT-003’s safety and tolerability profile continues to support a steroid-only prophylactic immunomodulation regimen.
  • Outpatient dosing has been enabled since September 2025.
  • No drug-induced liver injury (DILI), thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS) or myocarditis observed as of the January 9, 2026, cutoff date.
  • Cardiac safety monitoring has shown reductions in cardiac injury and early signals of cardiac systolic function normalization, as measured by left ventricular ejection fraction (LVEF) (N=14, data cutoff of September 29, 2025).
• Compelling microdystrophin expression levels (N=10, data cutoff of September 29, 2025) and concordant restoration of key components of the dystrophin-associated protein complex (DAPC) have been observed.
• Improvements across a range of biomarkers of muscle integrity have been observed suggesting a coordinated downstream effect (N=11-14, data cutoff of September 29, 2025).
• During the first half of 2026, the Company plans to have multiple interactions with the U.S. Food and Drug Administration (FDA) to align on its Phase 3 confirmatory trial design and on necessary confirmatory evidence required to support potential accelerated approval for SGT-003, with an update expected mid-year 2026.
• The Company expects to report additional data from the Phase 1/2 INSPIRE DUCHENNE trial in mid-2026.
  
  

Ex-U.S.:

• The first participant has been enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected to occur in the first quarter of 2026.
• The IMPACT DUCHENNE trial has two active clinical trial sites, located in Canada and Australia, with planned expansion into additional countries, including in Europe, beginning in mid-year 2026, subject to the receipt of regulatory approvals.
• SGT-003 has been awarded the Innovation Passport Designation under the new U.K. Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market and facilitate patient access to new medicines, positioning SGT-003 to potentially become the first-to-market Duchenne gene therapy in the U.K.
  
  

SGT-212 for Friedreich’s ataxia (FA)

• The first participant has been dosed in FALCON, a Phase 1b first-in-human clinical trial evaluating SGT-212, Solid’s investigational gene therapy for the treatment of FA.
  • Intra-procedural MRI-imaging demonstrated promising intradentate nuclei (IDN) targeting and coverage.
  • SGT-212 is the first investigational gene therapy for FA to utilize a dual route of administration and is intended to promote restoration of therapeutic levels of the frataxin protein to address the neurologic, cardiac and systemic clinical manifestations of FA.
  • SGT-212 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA.
• The Company anticipates reporting initial data from the FALCON trial in the second half of 2026, subject to participant enrollment.
  
  

Cardiac Pipeline
SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT)

• Clinical trial sites have been activated with participant screening underway for ARTEMIS, a first-in-human, open-label, Phase 1b clinical trial to evaluate the safety, tolerability and efficacy of SGT-501, Solid’s investigational gene therapy for the treatment of CPVT.
• SGT-501 is a novel, investigational gene therapy intended to achieve augmentation of CASQ2 protein levels to address the underlying ryanodine receptor (RYR2) instability and calcium dysregulation seen in CPVT. There are currently no FDA-approved treatments that address the underlying mechanisms of CPVT.
• The Company anticipates reporting initial safety data from the ARTEMIS trial in the second half of 2026, subject to participant enrollment.
  
  

Platform Technologies – Capsids
Solid continues to broaden access to its proprietary next-generation capsid, AAV-SLB101, designed with the goal of enhancing skeletal muscle and cardiac tropism with reduced biodistribution to the liver.

• Solid has executed 50+ agreements, including licenses, with corporations, institutions, and academic labs for the use of AAV-SLB101.
• Used in SGT-003, AAV-SLB101 has been generally well tolerated in the 33 participants dosed in the INSPIRE DUCHENNE trial as of January 9, 2026, and has shown compelling levels of vector transduction, protein expression (N=10, data cutoff of September 29, 2025), and reduced liver impact (N=14, data cutoff of September 29, 2025).

44^th Annual J.P. Morgan Healthcare Conference Webcast
Mr. Cumbo will present at the 44^th Annual J.P. Morgan Healthcare Conference today at 5:15 p.m. PT (8:15 p.m. ET). A live webcast of the presentation will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page.

About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic neuromuscular and cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.

Cautionary Note Regarding 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 goals, priorities and key clinical and preclinical milestones; strategies and expectations for the company’s SGT-003, SGT-212 and SGT-501 programs; expectations for additional site activations, planned enrollment, planned data announcements, planned regulatory interactions and the potential approval pathways for SGT-003; plans for data announcements for the clinical trial of SGT-212; timing of planned enrollment and data announcements for the clinical trial SGT-501; 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 company’s ability to advance SGT-003, SGT-212, SGT-501, SGT-601 and other preclinical programs, capsid libraries and other enabling technologies 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 and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; enroll patients in ongoing trials; activate clinical trial sites; replicate preliminary or interim data from clinicals trials in the final data of such trials; compete successfully with other companies that are seeking to develop Duchenne, FA, CPVT 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-212, SGT-501, SGT-601 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 Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com

Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com

FAQ

How many participants has Solid Biosciences dosed with SGT-003 as of Jan 9, 2026?

SGT-003 has been dosed in 33 participants in the Phase 1/2 INSPIRE trial as of 1/9/2026.

When is dosing expected to start in the ex-U.S. Phase 3 IMPACT Duchenne trial (SLDB)?

Dosing for IMPACT Duchenne is expected in Q1 2026, with initial sites in Canada and Australia.

What regulatory designations has SGT-212 (FA) received for SLDB?

SGT-212 has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA.

What is AAV-SLB101 and how widely is it licensed by Solid Biosciences (SLDB)?

AAV-SLB101 is Solid’s next-generation capsid designed for muscle and heart tropism; Solid has executed 50+ licenses and agreements for its use.

When does Solid Biosciences expect to report additional INSPIRE Duchenne data?

The company expects to report additional Phase 1/2 INSPIRE data in mid-2026, subject to analysis and cutoff timing.

What early safety signals were reported for SGT-003 in Duchenne trials (SLDB)?

As of the 1/9/2026 cutoff, no DILI, TMA, aHUS or myocarditis were observed and SGT-003 was generally well tolerated.