Weekly Quick Hits (Greater Philly) – Week of May 29, 2023

By Mark Terry
June 2, 2023

Funding, Awards and Collaborations

CHOP and More than 20 Institutions Create Open-Analysis Platform for Pediatric Brain Tumors

Researchers with Children’s Hospital of Philadelphia (CHOP), the Alex’s Lemonade Stand Foundation Childhood Cancer Data Lab, and more than 20 other institutions partnered to form an open-source, reproducible analysis platform for pediatric brain tumors. It will leverage thousands of genomically sequenced samples and help researchers identify initial findings about genetic variants associated with poorer outcomes to guide diagnoses and therapeutics.

New Products

B. Braun Launches DoseTrac Enterprise Infusion Management Software

Bethlehem, Pa.-based B. Braun Medical launched its next generation of infusion management software, DoseTrac Enterprise Infusion Management Software. In addition to offering a mix of real-time views and retrospective reporting capabilities, it can connect up to 40,000 pumps at an unlimited number of facilities with a single application.

Research Roundup

Palatin Presented Preclinical Data on Diabetic Nephropathy

Cranbury, NJ-based Palatin Technologies presented preclinical data on MC1R agonist PL8177. In a rat model of diabetic nephropathy, the compound protected against podocyte loss in a streptozotocin-induced rat model of diabetic nephropathy.

Penn Research: Nanobots to Fight Fungal Infections

Researchers at the University of Pennsylvania School of Dental Medicine and its School of Engineering and Applied Science engineered a microrobotic system that can target and eliminate fungal pathogens. The miniaturized robotic system uses catalytic nanoparticles, known as nanozymes, which were controlled with electromagnetic fields.

CHOP Research: IgA Fine-Tunes Interactions with Microbes

Researchers at Children’s Hospital of Philadelphia (CHOP) published research that IgA acts as “tuner” that regulates the number of microbes the body sees every day, controlling the systemic immune response to these commensal microbes, and limiting the development of systemic immune dysregulation. The research was published in Science Immunology.

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Penn Research: Using “Good Fat” to Combat Obesity and Clear Blood Glucose

Researchers with the Perelman School of Medicine at the University of Pennsylvania and University of Cambridge, Free University of Brussels and University of East Anglia published research in Science Advances that shows the potential for using “good fat” tissue to fight obesity and clear glucose from the blood. Using Krios G3i, a cryogenic electronic microscope at Penn, they viewed mitochondrial uncoupling protein 1 (UCP1), which allows fat tissue to burn off calories as heat. This provided new insights into how these brown fat cells might be leveraged for weight loss.

Regulatory and Advocacy

FDA Approves Blue Earth Diagnostics’ Posluma for Prostate Cancer

Blue Earth Diagnostics, with offices in Monroe Township, NJ, and Oxford, UK, announced the FDA approved its PSMA-targeted PET imaging agent, Posluma (flotufolastat F 18) injection for men with prostate cancer with suspected metastasis who are candidates for initial definitive therapy or with suspected recurrence based on elevated PSA level. It is the first and only FDA-approved, PSMA-targeted imaging agent developed with proprietary radiohybrid technology.

Janssen Submits NDA for Single Tablet Combo of Macitentan/Tadalafil for PAH

The Janssen Pharmaceutical Companies of Johnson & Johnson (Raritan, NJ) announced it had submitted a New Drug Application (NDA) to the FDA for a single tablet combination of macitentan 10mg and tadalafil 40mg for long-term treatment of pulmonary arterial hypertension (PAH). The application is built on data from the Phase III A DUE trial, which hit the primary endpoint, demonstrating the drug combination significantly improved pulmonary hemodynamics compared to macitentan and tadalafil monotherapies.

Rocket Pharma’s RP-A501 for Danon Disease Received Priority Medicines Designation in EU

Rocket Pharmaceuticals, based in Cranbury, NJ, reported the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to RP-A501, its AAV-based gene therapy for Danon Disease. Danon Disease is a fatal inherited cardiomyopathy with no curative therapies.

FDA Approves AstraZeneca and Merck’s Lynparza + Abiraterone for Prostate Cancer

The FDA approved AstraZeneca and Merck’s Lynparza (olparib) in combination with abiraterone and prednisone or prednisolone for adults with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer. AstraZeneca has U.S. offices in Wilmington, Del. The approval was based on a subgroup analysis of the Phase III PROpel trial.