University of Pennsylvania
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1740-11-14
- Employees
- 5K
- Market Cap
- -
- Website
- http://www.upenn.edu
Landmark VA Study Reveals Genomic Differences in Metastatic Prostate Cancer Across Racial Groups
• Researchers from Moffitt Cancer Center and partners analyzed genomic data from over 5,000 veterans with metastatic prostate cancer, finding significant biological differences between non-Hispanic Black and white patients.
• Non-Hispanic Black veterans showed higher rates of genomic alterations associated with immunotherapy benefit, while white veterans had more frequent mutations in DNA repair genes and androgen receptor pathways.
• Despite these biological differences, both groups demonstrated similar survival outcomes when given equal access to care, highlighting the potential of precision oncology to reduce healthcare disparities.
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.
Breakthrough: First Personalized mRNA-Based CRISPR Therapy Successfully Treats Infant with Rare Metabolic Disorder
• Aldevron and Integrated DNA Technologies have manufactured the world's first personalized CRISPR gene editing therapy for an infant with urea cycle disorder, completing the process in just six months—three times faster than standard timelines.
• The N of 1 therapy required development of a new guide RNA sequence, mRNA-encoded base editor, custom safety services, and lipid nanoparticle formulation, demonstrating a potential roadmap for treating other rare genetic diseases.
• Results published in The New England Journal of Medicine show successful treatment of an infant with neonatal-onset CPS1 deficiency, offering proof of concept for safe and effective personalized CRISPR therapies.
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.
FDA Clears IND for MavriX Bio's Gene Therapy for Angelman Syndrome, First-in-Human Trial to Begin in 2025
• MavriX Bio has received FDA clearance for its Investigational New Drug application for MVX-220, a novel AAV gene therapy designed to restore UBE3A gene function in Angelman syndrome patients.
• The ASCEND-AS Phase 1/2 clinical trial will evaluate MVX-220 in both adult and pediatric patients with various Angelman syndrome genotypes, with first patient dosing anticipated in the second half of 2025.
• Developed at the University of Pennsylvania with support from the Foundation for Angelman Syndrome Therapeutics (FAST), MVX-220 represents a potential single-treatment therapy for a condition that currently has no approved treatments.
Kennedy Mandates Placebo Testing for All New Vaccines, Potentially Impacting Fall COVID Boosters
• U.S. Health and Human Services Secretary Robert F. Kennedy Jr. has announced that all new vaccines must undergo placebo-controlled safety trials before approval, described as a "radical departure" from past practices.
• The mandate could significantly impact the availability of updated COVID-19 booster shots expected for fall, which have typically been authorized without human trials when targeting new viral strains.
• Kennedy simultaneously announced an initiative for the NIH to accelerate development of new vaccines for COVID, bird flu, and seasonal flu using technologies other than mRNA, reflecting his long-standing criticism of vaccine oversight.
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.
SEED Therapeutics Advances Dual Breakthrough Programs in Targeted Protein Degradation at AACR 2025
• SEED Therapeutics presented two breakthrough research programs at AACR 2025, showcasing advances in targeted protein degradation technology for cancer treatment.
• The company's novel RBM39 degrader demonstrated complete tumor regression in Ewing sarcoma models, with IND filing expected mid-2025 after receiving FDA Rare Pediatric Disease and Orphan Drug designations.
• Researchers discovered a dual PROTAC approach using two different E3 ligases that synergistically enhances KRAS G12D degradation and overcomes the "hook effect" limitation in traditional PROTAC therapies.
BRCA2 Mechanism Reveals Why PARP Inhibitors Work in Only Some Cancer Patients
• NYU Langone researchers have discovered that BRCA2 acts as a molecular shield preventing PARP1 from binding to DNA damage sites, explaining why PARP inhibitors are effective only in certain cancer patients.
• The study, published in Nature, used advanced single-molecule imaging to reveal how BRCA2 ensures RAD51 protein can access DNA repair sites instead of PARP1, preventing treatment-generated DNA breaks in resistant cancer cells.
• This breakthrough explains variable patient responses to PARP inhibitors like olaparib, which have shown effectiveness in pancreatic, prostate, breast, and ovarian cancers with BRCA mutations, pointing to the need for patient-specific tumor profiling.
Opus Genetics Advances LCA5 Gene Therapy Program with First Pediatric Patient Dosing and Promising Adult Data
• Opus Genetics has dosed the first pediatric patient in their Phase 1/2 trial of OPGx-LCA5 gene therapy for Leber congenital amaurosis, with initial data expected by Q3 2025.
• New 12-month data from the first three adult patients treated with OPGx-LCA5 confirms durability of positive responses observed at 6 months, with results to be presented at a medical conference in Q2 2025.
• The company has scheduled an FDA meeting in March 2025 to discuss Phase 3 trial design and registrational endpoints, marking a significant step toward potential therapeutic advancement.
NIH Study: Single Dose of MEDI8852 Antibody Shows Complete Protection Against H5N1 Avian Flu in Primates
• A groundbreaking NIH study demonstrates that a single dose of MEDI8852, a broadly neutralizing antibody developed by MedImmune/AstraZeneca, provides complete protection against H5N1 avian influenza in macaques.
• The antibody targets a stable portion of the influenza virus protein, offering extended protection that could last weeks after administration, making it a promising preventive tool for potential H5N1 outbreaks.
• All macaques pre-treated with MEDI8852 survived H5N1 exposure with minimal or no symptoms, while untreated control subjects developed severe or fatal illness.
Kymriah Receives EU Approval as First CAR-T Therapy for Follicular Lymphoma
• Novartis' Kymriah is the first CAR-T therapy approved in the EU for relapsed/refractory follicular lymphoma, offering a new option for patients after multiple treatments.
• The approval was based on the ELARA trial, demonstrating an 86% response rate and 69% complete response rate in patients treated with Kymriah.
• Kymriah provides durable treatment effects, with 87% of complete responders remaining in remission for at least nine months after initial response.
• This approval expands Kymriah's indications in the EU, adding to its existing approvals for diffuse large B-cell lymphoma and acute lymphoblastic leukemia.
CAR-Enhancers Show Promise in Prolonging Remission in CAR T-Cell Therapy
• Researchers at Dana-Farber Cancer Institute have developed CAR-enhancers (CAR-E) to extend the effectiveness of CAR T-cell therapy.
• CAR-E aims to prevent relapse by maintaining the activity of CAR T-cells and promoting the formation of memory cells.
• The new CAR-E therapy is set to begin a phase I clinical trial focusing on safety, dosage, and treatment schedule post CAR T-cell infusion.
• CAR T-cell therapy, while effective for blood cancers, faces challenges with relapse due to T-cell exhaustion.
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.
Patient Voice Amplification Critical for Clinical Trial Success: Industry Analysis Shows 85% Retention Failure Rate
• Clinical trials face significant challenges with over two-thirds failing to meet enrollment targets and 85% struggling with patient retention, highlighting the urgent need for better participant engagement.
• Technology-driven solutions, including mobile apps and secure messaging systems, are emerging as key tools for improving patient engagement and reducing dropout rates in clinical trials.
• Patient engagement initiatives can increase clinical trial value by $35 million in pre-phase II projects, equivalent to accelerating product launch by 2.5 years, while addressing diversity and accessibility challenges.
© Copyright 2025. All Rights Reserved by MedPath