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Solid Biosciences Announces Licensing Agreement with Kinea Bio for the Use of Proprietary Next-Generation Capsid AAV-SLB101

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Solid Biosciences (NASDAQ:SLDB) has entered into a non-exclusive worldwide licensing agreement with Kinea Bio for its proprietary next-generation capsid, AAV-SLB101. The agreement allows Kinea Bio to use AAV-SLB101 for developing KNA-155, a gene therapy targeting dysferlinopathy, a form of limb-girdle muscular dystrophy.

Under the agreement, Solid will receive an upfront payment and is eligible for development and sales milestones, plus tiered royalties on net sales. The AAV-SLB101 capsid has demonstrated enhanced muscle tropism and reduced liver biodistribution in preclinical studies and early clinical data from the ongoing Phase 1/2 INSPIRE DUCHENNE trial, where it has been well-tolerated in 15 participants as of August 12, 2025.

This marks another successful partnership for Solid's AAV-SLB101 platform, with over 25 agreements and licenses executed to date.

Solid Biosciences (NASDAQ:SLDB) ha stipulato un accordo di licenza mondiale non esclusivo con Kinea Bio per il suo capsidio di prossima generazione proprietario, AAV-SLB101. L'accordo consente a Kinea Bio di utilizzare AAV-SLB101 per sviluppare KNA-155, una terapia genica mirata alla dysferlinopatia, una forma di distrofia muscolare di spalla e bacino.

Secondo l'accordo, Solid riceverà un pagamento iniziale ed è idonea a ricevere traguardi di sviluppo e di vendita, oltre a royalties scalari sulle vendite nette. Il capsidio AAV-SLB101 ha mostrato una maggiore tropismo muscolare e una minore biodistribuzione al fegato in studi preclinici e nei primi dati clinici del trial di fase 1/2 INSPIRE DUCHENNE in corso, dove è stato ben tollerato in 15 partecipanti alla data dell'12 agosto 2025.

Questo segna un altro successo di partnership per la piattaforma AAV-SLB101 di Solid, con oltre 25 accordi e licenze finora.

Solid Biosciences (NASDAQ:SLDB) ha suscrito un acuerdo de licencia mundial no exclusivo con Kinea Bio para su capsídio de próxima generación propietario, AAV-SLB101. El acuerdo permite a Kinea Bio usar AAV-SLB101 para desarrollar KNA-155, una terapia génica dirigida a la dysferlinopatía, una forma de distrofia muscular de la cinturas.

Según el acuerdo, Solid recibirá un pago inicial y será elegible para hitos de desarrollo y ventas, además de regalías escalonadas sobre las ventas netas. El capsidio AAV-SLB101 ha mostrado un mayor tropismo muscular y una menor biodistribución al hígado en estudios preclínicos y en los primeros datos clínicos del ensayo de fase 1/2 INSPIRE DUCHENNE en curso, donde se ha tolerado bien en 15 participantes a fecha del 12 de agosto de 2025.

Esto marca otra asociación exitosa para la plataforma AAV-SLB101 de Solid, con más de 25 acuerdos y licencias realizados hasta la fecha.

Solid Biosciences (NASDAQ:SLDB)가 자체 차세대 캡시드인 AAV-SLB101에 대한 비배타적 전세계 라이선스 계약을 Kinea Bio와 체결했습니다. 이 계약은 Kinea Bio가 AAV-SLB101을 사용하여 dysferlinopathy를 표적으로 한 유전자 치료제 KNA-155를 개발하는 데 허용합니다. 이는 사지 근육병의 한 형태입니다.

계약에 따라 Solid는 선지급금을 받고 개발 및 판매 이정표를 받을 자격이 있으며, 순매출에 대한 단계별 로열티를 받습니다. AAV-SLB101 캡시드가 전임상 연구 및 진행 중인 1/2상 INSPIRE DUCHENNE 임상 초기 데이터에서 근육에 대한 표적화 증가 및 간 내 분포 감소를 보였으며, 현재까지 2025년 8월 12일 기준 15명 참여가 진행 중인 임상에서 잘 견디고 있습니다.

이번 계약은 Solid의 AAV-SLB101 플랫폼과의 또 다른 성공적인 파트너십을 의미하며, 지금까지 25건이 넘는 계약과 라이선스가 체결되었습니다.

Solid Biosciences (NASDAQ:SLDB) a conclu un accord de licence mondial non exclusif avec Kinea Bio pour son capside de prochaine génération propriétaire, AAV-SLB101. Cet accord permet à Kinea Bio d’utiliser AAV-SLB101 pour développer KNA-155, une thérapie génique ciblant la dysferlinopathie, une forme de myopathie par atteinte des ceintures.

Selon l’accord, Solid percevra un paiement initial et sera éligible à des jalons de développement et de vente, ainsi que des royalties échelonnées sur les ventes nettes. Le capsid AAV-SLB101 a démontré un tropisme musculaire accru et une biodistribution hépatique réduite dans des études précliniques et dans les premiers résultats cliniques de l’essai de phase 1/2 INSPIRE DUCHENNE en cours, où il a été bien toléré chez 15 participants au 12 août 2025.

Cela marque un autre partenariat réussi pour la plateforme AAV-SLB101 de Solid, avec plus de 25 accords et licences signés à ce jour.

Solid Biosciences (NASDAQ:SLDB) hat eine nicht exklusive weltweite Lizenzvereinbarung mit Kinea Bio für sein proprietäres Next-Generation-Capsid AAV-SLB101 getroffen. Die Vereinbarung ermöglicht es Kinea Bio, AAV-SLB101 zur Entwicklung von KNA-155 zu verwenden, einer Gentherapie, die Dysferlinopathie anspricht, eine Form der muskulo-skelettalen Muskeldystrophie.

Im Rahmen der Vereinbarung erhält Solid eine Vorauszahlung und ist berechtigt, Meilensteine in Entwicklung und Vertrieb sowie gestaffelte Tantiemen auf Nettoeinnahmen zu erhalten. Das AAV-SLB101-Capsid hat in präklinischen Studien und ersten klinischen Daten der laufenden Phase-1/2-INSPIRE-DUCHENNE-Studie eine verbesserte Muskel-Tropismus und eine verringerte Leber-Biodistribution gezeigt und wurde bis zum 12. August 2025 in 15 Teilnehmern gut vertragen.

Dies markiert eine weitere erfolgreiche Partnerschaft für Solid's AAV-SLB101-Plattform, mit über 25 Vereinbarungen und Lizenzen bis heute.

Solid Biosciences (NASDAQ:SLDB) وقعت اتفاقية ترخيص عالمية غير حصرية مع Kinea Bio لـ capsid الجيل التالي المملوك لها، AAV-SLB101. تسمح الاتفاقية لـ Kinea Bio باستخدام AAV-SLB101 لتطوير KNA-155، علاج جيني يستهدف Dysferlinopathy، وهو شكل من أشكال ضمور عضلي يمتد من الساقين إلى الكتفين.

وفقاً للاتفاق، ستتلقى Solid دفعة مقدمة وتكون مؤهلة للحصول على أهداف التطوير والمبيعات، بالإضافة إلى عوائد متدرجة على صافي المبيعات. أظهر capsid AAV-SLB101 Tropism عضلي محسن وتوزعاً كبدياً منخفضاً في الدراسات قبل السريرية والبيانات السريرية الأولية من تجربة المرحلة 1/2 INSPIRE DUCHENNE الجارية، حيث تم تحملها جيداً في 15 مشاركاً حتى 12 أغسطس 2025.

يمثل هذا شراكة ناجحة أخرى لمنصة AAV-SLB101 لدى Solid، مع أكثر من 25 اتفاقاً وتراخيص حتى تاريخه.

Solid Biosciences (NASDAQ:SLDB) 已与Kinea Bio就其自主的下一代外壳蛋白 AAV-SLB101 达成全球范围内的非独家许可协议。该协议允许 Kinea Bio 使用 AAV-SLB101 来开发 KNA-155,一种针对 dysferlinopathy 的基因治疗,属于肢带性肌营养不良的一种形式。

根据协议,Solid 将获得预付款,并有资格获得开发和销售里程碑,以及净销售额分级版税。AAV-SLB101 的外壳在前临床研究和正在进行的阶段1/2 INSPIRE DUCHENNE 临床试验的早期数据中显示出增强的肌肉嗜性和较低的肝分布,在截至 2025 年 8 月 12 日的 15 名参与者 中耐受良好。

这是 Solid 的 AAV-SLB101 平台又一次成功的伙伴关系,目前为止已经 签署超过25项协议和许可

Positive
  • AAV-SLB101 shows strong safety profile with good tolerance in 15 trial participants
  • Expanding commercial reach with over 25 partnership agreements executed
  • Potential for multiple revenue streams through upfront payments, milestones, and royalties
  • Clinical data demonstrates robust cardiac and skeletal muscle transduction
Negative
  • License agreement is non-exclusive, limiting potential competitive advantages
  • Specific financial terms and milestone values not disclosed

Insights

Solid's licensing of AAV-SLB101 capsid to Kinea Bio strengthens revenue potential while validating their gene therapy delivery platform.

Solid Biosciences has executed a strategic non-exclusive licensing deal for their proprietary AAV-SLB101 capsid with Kinea Bio, marking their 25th partnership agreement for this technology. This transaction creates multiple value streams for Solid: an immediate upfront payment, potential development and sales milestone payments, and ongoing tiered royalties on future net sales.

The deal's significance extends beyond immediate economics. Kinea's selection of AAV-SLB101 for their dysferlinopathy gene therapy program (KNA-155) provides third-party validation for Solid's capsid technology. This capsid was rationally designed with enhanced muscle tropism and reduced liver biodistribution—critical characteristics for effective muscle-targeting therapies. The emerging safety data from 15 patients in Solid's INSPIRE DUCHENNE trial further de-risks this technology platform.

For Solid, this represents a dual-track value creation strategy: they advance their internal pipeline while simultaneously generating revenue through technology licensing. This approach effectively distributes risk across multiple programs and partners while maximizing the return on their R&D investment in delivery technology.

For investors, this expanding network of partnerships creates a potential long-term royalty stream across multiple therapeutic programs. Each new licensing deal increases the probability that at least some of these programs will reach commercialization, potentially creating sustained revenue for Solid that extends beyond their internal pipeline success.

Solid's AAV-SLB101 capsid shows promising clinical safety profile while enabling expansion into additional rare muscle diseases.

The licensing of AAV-SLB101 to Kinea Bio represents significant technological validation in the gene therapy field. The capsid's key differentiating features—enhanced muscle tropism with reduced liver distribution—address two critical challenges in gene therapy development: delivering sufficient therapeutic payload to target tissues while minimizing off-target effects that can lead to toxicity.

The preliminary clinical safety data from 15 dosed patients in the INSPIRE DUCHENNE trial is particularly noteworthy. AAV capsid-related toxicities (including liver effects and immune responses) have historically created significant hurdles for gene therapy programs. The reported tolerability in human subjects provides early evidence that AAV-SLB101 may overcome some of these obstacles.

Kinea's application of this capsid to dysferlinopathy (LGMD2B/R2) is scientifically logical, as both Duchenne and dysferlinopathies affect skeletal muscle but require different genetic payloads. Their KNA-155 program employs a dual AAV approach, likely necessary because the dysferlin gene exceeds traditional AAV packaging capacity, requiring split delivery strategies.

The fact that Kinea Bio is advancing into IND-enabling studies suggests reasonable confidence in preclinical proof-of-concept data. The expansion of AAV-SLB101 into this additional indication demonstrates how platform technologies in gene therapy can potentially address multiple rare genetic disorders with similar delivery requirements but different therapeutic payloads.

- Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for dysferlin-related limb-girdle muscular dystrophy -

- Solid to receive an upfront payment and is eligible for certain development and sales milestones and tiered royalties on net sales -

- Solid continues to expand collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date -

CHARLESTOWN, Mass., Sept. 23, 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, today announced a non-exclusive worldwide license and collaboration agreement with Kinea Bio for the use of Solid’s proprietary, next-generation capsid, AAV-SLB101, for the development and commercialization of KNA-155, an investigational dual AAV gene therapy targeting dysferlinopathy, a form of limb-girdle muscular dystrophy type 2B/R2 (LGMD2B/R2).

Under the terms of the agreement, Solid granted Kinea Bio a non-exclusive worldwide license to utilize AAV-SLB101 as the delivery backbone for KNA-155, which is advancing into IND-enabling preclinical activities. In return, Solid receives an upfront fee and is eligible for additional payments upon the achievement of certain development and sales milestones and tiered royalties on net sales.

AAV-SLB101 is Solid’s rationally designed capsid developed for enhanced muscle tropism and reduced biodistribution to the liver. Robust cardiac and skeletal muscle transduction and biodistribution have been demonstrated in preclinical studies as well as in early clinical data from Solid’s ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial (NCT06138639) evaluating SGT-003 (which utilizes AAV-SLB101), an investigational gene therapy to treat Duchenne muscular dystrophy. As of a data cutoff of August 12, 2025, AAV-SLB101 has been well tolerated in the 15 participants who have been dosed in the INPSIRE DUCHENNE trial.

“We are pleased to partner with the Kinea Bio team to expand AAV-SLB101’s application into dysferlin-related LGMD,” said Bo Cumbo, President and CEO of Solid Biosciences. “As we progress the INSPIRE DUCHENNE trial, the first-in-human evaluation of this next-generation capsid, we remain highly encouraged by the emerging safety profile, which we believe continues to de-risk and build the value proposition of AAV-SLB101 as a differentiated, muscle tropic capsid.

“At Solid, we are wholly focused on shaping a brighter future for gene therapy by creating the next wave of delivery tools, including capsids, promoters and CMC technology, all of which are developing into a rich constellation of synergistic tools that we believe will power future generations of gene therapies for patients,” Mr. Cumbo concluded.

“Solid’s next-generation capsid, AAV-SLB101, contributes critical, cutting-edge technology that we believe will provide a powerful delivery mechanism for our KNA-155 program,” said Casey Childers, CEO of Kinea Bio. “The robust preclinical and clinical data generated to date for AAV-SLB101 give us confidence in our potential to bring a safe and effective therapy to patients living with dysferlinopathies, and we look forward to partnering with the highly experienced team at Solid as we advance development.”

About AAV-SLB101
AAV-SLB101 is a proprietary, rationally designed capsid developed for enhanced muscle tropism and reduced liver uptake. With a robust preclinical package in mice and nonhuman primates, AAV-SLB101 has demonstrated increased transduction speed, enhanced skeletal and cardiac muscle tropism, decreased liver biodistribution and improved efficiency when compared to first generation capsids. The incorporation of AAV-SLB101 into AAV delivered therapies has the potential to be a step forward in the treatment of neuromuscular and cardiac diseases. Solid Biosciences aims to license AAV-SLB101 broadly to both companies and academic institutions pursuing treatments for rare diseases. Solid has existing licensing agreements with more than 25 academic labs, institutions, and companies for the use of AAV-SLB101.

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 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.

About Kinea Bio, Inc.
Kinea Bio, Inc. is a biotechnology company pioneering a novel dual AAV vector platform SIMPLI-GTTM to deliver large therapeutic genes that exceed the natural packaging capacity of AAV. The company was among the first to demonstrate the potential of this approach in systemic disorders such as Duchenne muscular dystrophy and is now expanding its pipeline to include dysferlinopathy (LGMD2B/R2) and other severe genetic conditions. Through innovative science and strategic collaborations, Kinea Bio is dedicated to translating breakthrough biology into transformative medicines for patients with high unmet needs. For more information, please visit www.kineabio.com.

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 achieve key clinical milestones; the Company’s pipeline of capsid products, including SLB-101, and programs for neuromuscular and cardiac diseases, including its SGT-501, SGT-212 and SGT-003 programs and expectations for CTA filings, site activations, clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; 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 company’s ability to advance and license AAV-SLB101 and advance SGT-212, SGT-003, SGT-501, SGT-601, SGT-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 and early-stage clinical trials 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, Friedreich’s ataxia 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-212, SGT-003, SGT-501, SGT-601, SGT-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 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

What is the licensing agreement between Solid Biosciences (NASDAQ:SLDB) and Kinea Bio?

Solid Biosciences has granted Kinea Bio a non-exclusive worldwide license to use its AAV-SLB101 capsid for developing KNA-155, a gene therapy targeting dysferlinopathy. The agreement includes upfront payments, milestone payments, and tiered royalties.

What are the clinical results of AAV-SLB101 in Solid Biosciences' trials?

As of August 12, 2025, AAV-SLB101 has been well-tolerated in 15 participants in the Phase 1/2 INSPIRE DUCHENNE trial, showing robust cardiac and skeletal muscle transduction and reduced liver biodistribution.

How many partnership agreements has Solid Biosciences secured for AAV-SLB101?

Solid Biosciences has executed more than 25 agreements and licenses for its AAV-SLB101 technology platform.

What is AAV-SLB101 and what makes it unique?

AAV-SLB101 is Solid's proprietary, rationally designed capsid developed for enhanced muscle tropism and reduced biodistribution to the liver, making it particularly suitable for muscle-targeted gene therapies.

What disease will Kinea Bio target using Solid Biosciences' AAV-SLB101?

Kinea Bio will use AAV-SLB101 to develop KNA-155, a gene therapy targeting dysferlinopathy, a form of limb-girdle muscular dystrophy type 2B/R2 (LGMD2B/R2).
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