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CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB), a biotechnology company with a market capitalization of $402 million, has entered a non-exclusive worldwide license agreement with Kinea Bio for its proprietary AAV-SLB101 capsid, according to a press release statement issued Tuesday. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet, though it faces challenges with rapid cash burn.
The agreement allows Kinea Bio to use AAV-SLB101 as the delivery backbone for KNA-155, an investigational dual AAV gene therapy targeting dysferlinopathy, a form of limb-girdle muscular dystrophy type 2B/R2. KNA-155 is currently advancing into IND-enabling preclinical activities.
Under the terms of the agreement, Solid Biosciences will receive an upfront payment and is eligible for additional payments upon reaching certain development and sales milestones, plus tiered royalties on net sales.
AAV-SLB101 is designed for enhanced muscle tropism and reduced liver uptake. The capsid is currently being evaluated in Solid’s Phase 1/2 INSPIRE DUCHENNE clinical trial for SGT-003, a gene therapy for Duchenne muscular dystrophy. As of August 12, the capsid has been well tolerated in 15 participants who have been dosed in the 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. For detailed analysis and additional insights about Solid Biosciences’ financial health and growth prospects, investors can access comprehensive research reports on InvestingPro, which currently indicates the stock may be undervalued based on its Fair Value analysis.
Casey Childers, CEO of Kinea Bio, stated that AAV-SLB101 "contributes critical, cutting-edge technology that we believe will provide a powerful delivery mechanism for our KNA-155 program."
This marks the latest in a series of collaborative efforts for Solid’s AAV-SLB101 technology, with the company having executed more than 25 agreements and licenses to date with academic labs, institutions, and companies.
Solid Biosciences focuses on developing gene therapy candidates for rare neuromuscular and cardiac diseases, while Kinea Bio specializes in dual AAV vector platforms for delivering large therapeutic genes.
In other recent news, Solid Biosciences has received a Fast Track designation from the U.S. Food and Drug Administration for its SGT-501 gene therapy, aimed at treating catecholaminergic polymorphic ventricular tachycardia (CPVT). This designation is in addition to the Orphan Drug and Rare Pediatric Disease designations the therapy has already received, potentially allowing for more frequent FDA interactions and priority review. Meanwhile, Cantor Fitzgerald has reiterated its Overweight rating with a $16.00 price target on Solid Biosciences, highlighting the company’s Duchenne muscular dystrophy (DMD) program’s novel approach that targets muscle tissue effectively. Similarly, JMP Securities has reaffirmed its Market Outperform rating and a $15.00 price target, citing advancements in Solid Biosciences’ gene therapy programs, including those for DMD, Friedreich’s Ataxia, and CPVT. Notably, Solid Biosciences has dosed 15 DMD patients ahead of schedule and plans to meet with the FDA in the fourth quarter of 2025 to discuss regulatory pathways for its SGT-003 therapy. These developments underscore the company’s ongoing efforts in advancing its therapeutic programs.
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