From FDA to Directly Proceed to Registrational Trial for Approval of Ryoncil® in Duchenne Muscular Dystrophy - CORRECTION

April 13, 2026

Working with Parent Project Muscular Dystrophy and the Duchenne Registry on patient identification and trial awareness efforts 


~15,000 children are living with DMD in the U.S.

NEW YORK, April 10, 2026 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to directly proceed for a registrational clinical trial evaluating Ryoncil® (remestemcel-L-rknd) in Duchenne muscular dystrophy (DMD), which affects approximately 15,000 children in the U.S.1


Ryoncil® is the first mesenchymal stromal cell (MSC) product approved by FDA, and the only product approved for children under age 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD)2. This new registrational trial builds on Ryoncil’s® proven safety in children, evidence of efficacy in DMD preclinical models, and FDA-approved manufacturing process. Leveraging Ryoncil's® anti-inflammatory mechanism of action in SR-aGvHD, Mesoblast aims to reduce the inflammatory cascade characteristic of DMD, preserve muscle function, and slow disease progression.


The trial will randomize 76 patients aged 5 to 9 years to either Ryoncil® (7 infusions of 2 x 106 cells/kg over 9 months) or placebo, on top of standard of care. The trial’s primary endpoint will be time-to-stand at nine months, a validated FDA endpoint for approval. To help support successful execution of this study, Mesoblast has contracted with Parent Project Muscular Dystrophy (PPMD) and the Duchenne Registry to support patient identification and trial awareness.


“This study represents an important step forward in potentially addressing the inflammatory component of DMD, a major driver of disease progression,” said Aravindhan Veerapandiyan, MD, Director of the Comprehensive Neuromuscular Program at Arkansas Children’s Hospital, and Principal Investigator of the study. “By leveraging the anti-inflammatory effects of Ryoncil, we aim to intervene at a stage where muscle tissue may still be preserved, potentially altering the trajectory of the disease.”


“We are very pleased to have received clearance to proceed directly to a registrational study for DMD based on our preclinical data in DMD animal models and our extensive safety data in children with SR-aGvHD. Our experience with Ryoncil suggests that we may have a unique approach to help with this devastating disease in children,” said Silviu Itescu, Chief Executive of Mesoblast

This press release is a correction to version dated April 7, 2026, which was not intended to imply any endorsement by PPMD.


About Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the muscle leads to loss of ambulation, respiratory failure and cardiomyopathy ultimately leading to death by the third decade.


About Parent Project Muscular Dystrophy
Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.


We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals.


Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.


About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast’s Ryoncil® (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.


Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil® is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.


About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2044 in all major markets.


About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.


Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast


References / Footnotes

  1. https://cureduchenne.org/about/what-is-duchenne/#:~:text=Prevalence,Cardiac%20Dysfunction
  2. Please see the full Prescribing Information at www.ryoncil.com

Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.


Release authorized by the Chief Executive.

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Duchenne muscular dystrophy won't stop NJ teen from learning to drive

June 26, 2026
MANALAPAN -- Jamesy Raffone turned 17 in March, and like most kids his age, he’s eager to get his driver’s license. “It’ll be cool, the freedom of it,” he said. His journey to that freedom, though, contains a lot more twists and turns than the typical teen’s. Jamesy has Duchenne muscular dystrophy, a genetic condition that results in a progressive loss of strength and eventually leads to paralysis and fatal heart and lung problems. When he was diagnosed, at age 4, a geneticist told Jamesy’s parents driving would be impossible. That is no longer the case. At Howell High School, as a junior taking driver’s education, Jamesy passed his written permit test earlier this year. “It wasn’t hard,” he said. Now comes the harder part: Driving lessons with an accessible vehicle that is outfitted with custom-fit hand controls so Jamesy, who gets around in a motorized wheelchair, does not need his legs to brake or accelerate. It’ll be costly, time-consuming and rigorous — the kind of challenge the Raffones have taken on time and again over the years through their nonprofit JAR of Hope, which helps Duchenne families. “By getting his license after being told he’ll never do it, he’s defying the odds and leading the way for other kids like him,” dad Jim Raffone said. “We want to be able to show them, those kids, that’s it’s possible — you can do this.” Beating expectations At the time of Jamesy’s diagnosis, the median life expectancy of a Duchenne patient was 23. Now it’s close to 30. Even by the improved benchmarks, Jamesy is doing well. He receives a stem-cell infusion every 45 days. “He’s head and shoulders above where he could be or should be for the milestones of a child with Duchenne,” Jim Raffone said. “He can sit upright — no scoliosis. His heart is good. He still has movement in his upper girdle (his arms and torso), which is amazing at this stage.” Keeping that movement is the key to being able to operate the joysticks that drive a specially outfitted car. In order to figure out which hand controls work best for Jamesy and instruct him on their use, the Raffones are working with Brant’s Driving School in Western Pennsylvania, which specializes in adaptive training. After a test run with a Brant vehicle at their Manalapan home, Jamesy and mom Karen Raffone are heading out there in July for a three-week course. Then they’ll have to get those controls installed in their own van. The whole enterprise could cost upwards of $40,000. “If we didn’t have the support of JAR of Hope, what would we do?” Jim Raffone said. “I don’t think he’d be able to drive.” Jim built the charity through years of audacious initiatives, including assembling the world’s longest Lego chain , hiking to Mount Everest base camp , ringing the closing bell of the New York Stock Exchange , and running a series of ultramarathons. In early May, despite three herniated discs in his neck suffered in a car accident, Jim completed the Mingus Traverse — an 82-mile race through Arizona desert and mountains. He crossed the line 117th out of 118 finishers in a time of 43 hours and 15 minutes. “It was so grueling,” he said. Then he underwent surgery. 'My son has taught me a lot' In Raffone’s garage is a motorized wheelchair JAR of Hope purchased for a family in Texas. Jim plans on delivering it personally in late June. “Sometime after Father’s Day,” he said. Father’s Day carries deep meaning for this family. When Jim first ventured into Duchenne advocacy, he said, moms were doing most of the heavy lifting. By his count, there are now eight dads who branched off of JAR of Hope to start their own initiatives. “They’re all raising a tremendous amount of money for the community,” he said. “It’s a second degree from JAR of Hope, and it’s very flattering. To give them that inspiration, that feeling that they can go out there and do this too, it’s pretty awesome.” Jim’s inspiration comes from within his own household. “My son has taught me a lot about resiliency,” he said. Driving was supposed to be out of reach for Jamesy. Who knows what barrier he’ll bust through next. “I always tell people, ‘Never give up,’” his dad said. “You have to keep pushing. The cure, or something to slow down the disease, could come at any time.” For more information on JAR of Hope, visit www.jarofhope.org . Jerry Carino is community columnist for the Asbury Park Press, focusing on the Jersey Shore’s interesting people, inspiring stories and pressing issues. Contact him at jcarino@gannettnj.com .

Current DMD Treatments Should Be Built For All Mutations

June 26, 2026
Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene. Not one mutation. Hundreds of them. These different mutations can disable the gene, each occurring in a different part of the DNA sequence. The mutation a patient carries determines which treatments they are eligible for. Exon-skipping drugs, the most widely prescribed category of DMD treatment, work by prompting the body to produce a partial dystrophin by reading around the damaged section of the gene. Each drug targets a specific exon, and each exon covers only a specific subset of patients. Sarepta's approved exon-skipping portfolio includes three distinct drugs, each addressing a different exon group and each serving a different slice of the DMD population. But a patient whose mutation falls outside those covered groups has no approved exon-skipping option. Full-length gene replacement delivers a complete, functional copy of the dystrophin gene rather than prompting the body to produce a partial one. That doesn’t depend on specific mutations. Instead, it’s being developed as a mutation-agnostic gene replacement approach. The platform has not yet been tested in humans, and all current data comes from preclinical animal models. Myosana Therapeutics’ design represents a new category of approach: mutation-agnostic, beyond any requirement to match a specific mutation to a specific drug. The company believes this design may have broader applicability across mutation types, though that has not been evaluated in human studies. Invest in Myosana Therapeutics on Wefunder This offering is made under Regulation CF. Investments are speculative, illiquid, and involve a high degree of risk. You should not invest unless you can afford to lose your entire investment. Please review all offering materials on Wefunder before investing in Myosana Therapeutics.

Understanding the FDA’s New ELEVIDYS Warning: A Family-Centered Perspective

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