Local 7-year-old gets ‘new lease on life' after new gene therapy
Lucas Solano was diagnosed with an incurable muscle disease at a young age. Something as simple as walking up stairs wasn't possible for Salano before the therapy.
'When he was a baby, he was a toddler: completely normal, he would climb up the stairs, he crawled, he walked within normal age range, and then all of a sudden it was like he couldn't play outside, he would ask for help to get upstairs,' Mother Maria Solano said.
Maria says her son was diagnosed with a muscle deterioration disease called Duchenne Muscular Dystrophy.
ALSO READ: Atrium Health gives free health screenings to 2,000 student athletes
However, Lucas' life changed about six months ago. He was the first at Atrium Health Levine Children's Hospital to receive the new gene therapy that produces a missing protein that helps build muscle.
It's changed his life completely.
'He has a whole new lease on life,' Maria Solano said.
The therapy is all about building muscle strength.
'The whole idea is that we are able to improve their muscle strength and be able to continue to walk for a longer period of time,' Dr. Urvi Desai said.
Desai said children diagnosed with the disease can lose their ability to walk in their teens and die young and says the new treatment is allowing Lucas to live his best life, doing things he loves.
'There's always hope and he's a testament to that, Sergio Solano, Lucas' father, said.
Lucas received treatment in December. Atrium Health says since then, 5 more children have received it.
Atrium said three more children are set to get the therapy in August.
(WATCH BELOW: Atrium Health launches home care program for pediatric patients)
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
CNBC
2 days ago
- CNBC
Sarepta resumes shipping of gene therapy Elevidys to patients who can walk
Sarepta Therapeutics said on Monday it will resume shipping of its gene therapy Elevidys to patients with a rare muscular disorder who can walk, after the FDA recommended the removal of a voluntary hold that was placed on the therapy. However, the use of the gene therapy remains on hold for Duchenne Muscular Dystrophy patients who cannot walk, the FDA said, adding that it is continuing to work with the company while investigating the death of two patients. The FDA's recommendation for ambulatory patients — those who can walk — followed a probe that showed the death of an eight-year-old boy in Brazil, was unrelated to the gene therapy, the agency said. Roche, which has partnered with Sarepta for commercialization outside the U.S., had previously said the patient's death was not related to the therapy, according to the reporting physician's assessment. Sarepta is also facing intense scrutiny following the death of two non-ambulatory teenage boys associated with Elevidys, as well as a 51-year-old man who had received its experimental gene therapy SRP-9004.
Medscape
5 days ago
- Medscape
EMA Says No to Duchenne Gene Therapy Elevidys
At its July 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use recommended not granting marketing authorization for the Duchenne muscular dystrophy (DMD) gene therapy treatment delandistrogene moxeparvovec (Elevidys, Sarepta Therapeutics). Patients with DMD lack normal dystrophin, found primarily in skeletal and cardiac muscle cells, such that their muscles become progressively weaker and eventually stop working. Elevidys is an adeno-associated virus vector-based gene therapy product for ambulatory patients aged 4 years and older with a confirmed mutation in the DMD gene. It contains the active substance delandistrogene moxeparvovec, made of a virus containing genetic material, to produce a truncated version of dystrophin and thereby slow down disease progression. It is given as a single infusion as a one-time treatment designed to treat the underlying cause of DMD. In making their decision, the EMA said that a study had failed to show that Elevidys had an effect on movement abilities after 12 months. No Significant Improvement The study involved 125 children aged between 4 and 7 years with DMD who were able to walk and who received one infusion of either delandistrogene moxeparvovec or placebo. The main measure of effectiveness was an effect on movement abilities over 12 months, assessed using the North Star Ambulatory Assessment (NSAA). The scale ranges from 0 to 34, with higher scores indicating better movement abilities. Improvements in NSAA scores were observed in patients who received delandistrogene moxeparvovec and placebo. The difference in the change in scores between the two groups was 0.65, which was not statistically significant. In addition, although many patients treated with delandistrogene moxeparvovec were shown to produce a shorter form of the dystrophin protein, the levels of dystrophin could not be linked to an improvement in movement abilities, the EMA said. Acute Liver Failure Deaths In March this year, a 16-year-old boy died from acute liver failure after receiving treatment with Elevidys in December the previous year. In the wake of this — and at the request of the EMA — in April this year, the company temporarily halted clinical studies of the treatment in the EU. Patients who had previously been treated with Elevidys in a clinical trial continued to be monitored. In June, a second patient, aged 15, who had been treated with the drug also died from acute liver failure. Earlier this month, the FDA asked the company to include a "black-box" warning for the risk of acute liver injury and liver failure in patients with DMD who can walk. The EMA said that the company that had applied for a marketing authorization may ask for re-examination of the opinion within 15 days of receiving the agency's opinion. Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.
Yahoo
23-07-2025
- Yahoo
Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
DURHAM, N.C., July 23, 2025--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). "Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy," said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. "This regulatory milestone builds on our recent receipt of Rare Pediatric Disease designation and, together with our preclinical body of evidence, gives us tremendous confidence as we move this program towards the clinic. Looking ahead, we remain in active dialogue with the FDA as we advance PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026." The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides sponsors certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for that use. About PBGENE-DMD PBGENE-DMD is Precision's development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near full-length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address up to 60% of the DMD patient population. In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body's ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement. PBGENE-DMD was recently granted FDA Rare Pediatric Disease (RPD) designation for the treatment of DMD. The Company is advancing the final IND-enabling toxicology studies and is working closely with leading DMD clinicians to design a first in human trial optimized for safety and efficacy. Clinical grade material is in preparation, with initial clinical data expected in 2026. About Precision BioSciences, Inc. Precision's two lead programs, PBGENE-HBV, for chronic Hepatitis B, and PBGENE-DMD, for Duchenne muscular dystrophy, are focused on areas with large patient populations with high unmet need. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline prioritizes in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners' and licensees' product candidates and gene editing approaches; the potential of PBGENE-DMD to safely drive meaningful improvement in functional and durable benefit over time for up to 60% of patients with DMD; the design on PBGENE-DMD to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein proven known to be functional in humans; the benefits of an Orphan Drug Designation including financial incentives and regulatory exclusivity; the potential value of a Priority Review Voucher(if awarded) including priority review for a different product or sale to another sponsor; the expected timing of regulatory processes and clinical operations (including IND and/or CTA filings, studies, enrollment and clinical data for PBGENE-DMD; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "appear," "approach," "believe," , "confidence", "contemplate," "could," "design" "designed," "estimate," "expect," "goal," "intend," "look," "may," "mission," "plan," "possible," "potential," "predict," "project," "pursue," "should," "strive," "suggest," "target," "will," "would," or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. View source version on Contacts Investor and Media Contact: Naresh TannaVice President of Investor
