CHILDREN'S HOSPITAL OF PHILADELPHIA
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Established
- 1855-01-01
- Employees
- -
- Market Cap
- -
- Website
- http://www.chop.edu/
CHOP Researchers Develop Breakthrough AAV Vectors for Brain Gene Therapy at Lower Doses
• Researchers at Children's Hospital of Philadelphia have engineered new adeno-associated viral (AAV) vectors that can target brain cells at significantly lower doses than current therapies, potentially improving safety and reducing costs.
• Two novel capsids were identified: AAV-Ep+ for Batten disease treatment and AAV-DB-3 for Huntington's disease, both showing robust efficacy in preclinical models and human neurons derived from stem cells.
• The breakthrough could transform treatment for neurodegenerative disorders by enabling one-time precision therapies that require lower doses while maintaining therapeutic efficacy, with potential applications for other inherited disorders.
Acuitas Therapeutics and CHOP Pioneer Personalized CRISPR Therapy for Infant with Urea Cycle Disorder
• Acuitas Therapeutics, Children's Hospital of Philadelphia, and University of Pennsylvania successfully delivered the world's first personalized LNP-delivered CRISPR gene-editing therapy to an infant with urea cycle disorder, with no adverse events reported.
• The groundbreaking therapy was developed, manufactured, and delivered in just six months, establishing a new model for rapid development of personalized gene therapies through cross-functional partnerships.
• Acuitas also presented advances in targeted LNP delivery, including DARPin-conjugated formulations achieving up to 98% binding and 90% expression in human CD8+ T cells, expanding therapeutic applications beyond the liver.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
CHOP Researchers Propose ALK Inhibitors as New Standard for Hereditary Neuroblastoma
• A groundbreaking case report from Children's Hospital of Philadelphia demonstrates successful long-term remission in hereditary neuroblastoma patients using targeted ALK inhibitors, potentially transforming treatment protocols.
• Researchers documented complete response in both a mother and daughter carrying the ALK R1275Q mutation, suggesting ALK inhibitors could replace intensive chemotherapy as frontline therapy for this genetic subtype.
• The study challenges current surveillance guidelines by recommending lifelong monitoring for hereditary neuroblastoma patients, as the mother was diagnosed at age 36 despite being asymptomatic.
FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval
• The FDA has asked Novavax to conduct an additional randomized, controlled study of its COVID-19 vaccine NVX-CoV2601 before considering full approval, despite the vaccine having emergency use authorization since 2022.
• Interim results from a phase 2/3 study showed Novavax's updated XBB.1.5 variant vaccine generated 5.8 times higher neutralizing antibody levels compared to the original formulation, with a favorable safety profile.
• The FDA's request represents a significant setback for Novavax, which had expected approval based on prior conversations with regulators and had already passed its April 1 PDUFA date.
FDA Expands Luminopia's Digital Therapeutic Approval for Amblyopia Treatment to Children Aged 8-12
• Luminopia's VR-based digital therapeutic for amblyopia has received FDA clearance for children aged 8-12 years, expanding from its previous 4-7 year age indication based on robust real-world evidence.
• The treatment offers a novel binocular approach using modified TV content through VR headsets, showing comparable efficacy in pre-teens as in younger children—the first such approval for this age group in over 20 years.
• Patients previously treated with traditional therapies for an average of 1.8 years still gained an additional line of vision when switching to Luminopia, addressing a significant unmet need for approximately 400,000 older children with amblyopia.
ADHD Persists in Adults with Autism at Rates Similar to Childhood, Study Finds
• New research reveals that ADHD rates among adults with autism remain nearly identical to those seen in children, affecting approximately 27% of autistic adults without intellectual disabilities.
• The co-occurrence of ADHD with autism is associated with poorer health outcomes, including higher rates of substance use, heart conditions, and injuries, according to data from over 3.5 million Medicaid enrollees.
• Despite the high prevalence, fewer than 50% of affected adults receive ADHD medication, though treatment appears to significantly reduce associated physical health complications.
Healx Initiates Phase 2 Trial of AI-Discovered HLX-1502 for Neurofibromatosis Type 1 Treatment
• Healx has dosed the first patient in INSPIRE-NF1, a Phase 2 trial evaluating HLX-1502, an oral therapy discovered through AI technology, for treating neurofibromatosis type 1.
• The investigational drug HLX-1502 has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, highlighting its potential as an alternative to current MEK inhibitors.
• The trial will enroll approximately 20 patients with plexiform neurofibromas across U.S. centers, focusing on tumor response rate, safety, and pharmacokinetics.
Large-Scale Study Confirms Safety of CAR T-Cell Therapy: No Link to Secondary Cancers Found
• A comprehensive study of 783 patients published in Nature Medicine found no evidence that CAR T-cell therapy causes secondary cancers through insertional mutagenesis.
• Researchers identified 18 cases of secondary cancers post-treatment, attributing them to prior chemotherapy and radiation rather than CAR T-cell therapy itself.
• The findings provide crucial safety validation for CAR T-cell therapy, which has treated over 30,000 patients with hematologic malignancies since its first approval in 2017.
FDA Greenlights Clinical Trials for Pig-to-Human Kidney Transplants
• The FDA has approved groundbreaking clinical trials for two biotech companies to transplant genetically modified pig kidneys into patients with kidney failure.
• The innovative approach aims to address the critical organ shortage affecting thousands of Americans currently waiting for kidney transplants.
• This development represents a significant milestone in xenotransplantation research, potentially offering new hope for patients with end-stage renal disease.
Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years
• Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B.
• Patients treated with Hemgenix experienced a 90% reduction in adjusted annualized bleeding rate (ABR) at year four compared to the lead-in period.
• 94% of patients remained free of continuous prophylaxis treatment at year four, indicating sustained independence from regular infusions.
• The gene therapy maintained a favorable safety profile, with most adverse events occurring within the first six months post-treatment.
TCL1A Protein Identified as Predictor of Blinatumomab Response in Relapsed B-ALL
A study presented at the American Society of Hematology annual meeting has identified TCL1A as a potential predictive biomarker for blinatumomab response in relapsed pediatric B-cell acute lymphoblastic leukemia (B-ALL), offering new insights into improving treatment strategies.
FDA Approves Ozempic for Reducing Kidney Disease Risks in Type 2 Diabetes Patients
• The FDA has approved Ozempic to reduce the risk of kidney disease worsening, kidney failure, and cardiovascular death in adults with type 2 diabetes and chronic kidney disease.
• A Phase 3 clinical trial demonstrated a 24% reduction in the risk of kidney disease progression and cardiovascular death compared to placebo with Ozempic.
• Ozempic is now the most broadly indicated GLP-1 receptor agonist, offering a new treatment option for millions of adults with type 2 diabetes and chronic kidney disease.
• The approval was based on the FLOW trial, which showed significant benefits in kidney and cardiovascular outcomes, leading to early termination due to efficacy.
First-of-its-kind Gene Editing Treatment Saves Baby with Rare Genetic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with CPS1 deficiency using a custom-designed CRISPR base editing therapy, marking a groundbreaking advancement in personalized genetic medicine.
• The experimental treatment corrected a specific genetic mutation in KJ Muldoon's DNA, allowing his liver to properly process ammonia and preventing toxic buildup that threatens brain development and survival.
• Researchers believe this pioneering approach could transform treatment for millions with rare genetic disorders, demonstrating that personalized gene therapies can be developed relatively quickly and at costs comparable to conventional treatments.
Clinical Trials Market to Reach $85.37 Billion by 2033, Driven by Globalization and Precision Medicine
• The clinical trials market is projected to reach USD 85.37 billion by 2033, driven by the increasing globalization of clinical trials and the need to access diverse patient populations.
• The expansion of precision medicine is creating significant opportunities for clinical trials focused on validating targeted therapies and identifying relevant biomarkers.
• North America currently dominates the clinical trials market due to its robust healthcare infrastructure and favorable regulatory environment, with Europe showing strong growth.
• Recent developments include FDA approval of Spinogenix's ALS trial and the NHS launch of a platform to accelerate personalized cancer vaccine clinical trials.
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