JOHNS HOPKINS UNIVERSITY

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

7 days ago

• 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.

Maryland Stem Cell Research Fund Allocates $18 Million to Advance Regenerative Medicine Research

9 days ago

• The Maryland Stem Cell Research Commission has awarded over $18 million in grants to 52 investigators working on nearly 50 different medical conditions including sickle cell anemia, diabetes, and various neurological disorders. • Funding recipients include innovative companies like Seraxis Inc. and Britecyte Inc., alongside researchers from major Maryland institutions, with the University of Maryland Eastern Shore receiving its first-ever stem cell research grant. • Since its establishment in 2006, the MSCRF has invested over $200 million in more than 650 projects, generating $525 million in economic activity and creating over 2,000 jobs across Maryland.

FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval

a month ago

• 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 to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs

a month ago

• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation. • The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data. • This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.

Northwestern Medicine Launches Landmark Study Using Apple Watch to Revolutionize AFib Treatment

2 months ago

• Northwestern Medicine researchers are initiating a seven-year clinical trial to determine if wearable technology can reduce continuous blood thinner use in atrial fibrillation patients, potentially decreasing bleeding risks and improving quality of life. • The REACT-AF trial will monitor participants using Apple Watch technology, implementing a "pill-in-pocket" approach where patients take anticoagulants only during detected AFib episodes rather than daily for life. • With AFib affecting over 5 million Americans and expected to reach 12.1 million by 2030, this NIH-funded study could fundamentally change treatment standards by personalizing anticoagulation therapy based on real-time heart rhythm monitoring.

Belay Diagnostics and GenomOncology Partner to Enhance CNS Cancer Liquid Biopsy Testing

2 months ago

• Belay Diagnostics has integrated GenomOncology's Pathology Workbench into its workflow to optimize analysis of its Summit™ liquid biopsy test for central nervous system malignancies. • The partnership enables efficient processing of thousands of samples monthly, reducing laboratory overhead and accelerating turnaround time from 7-10 days, providing faster treatment recommendations for CNS cancer patients. • Belay's Summit™ test detects genetic variants in CSF samples, while their complementary Vantage™ test assesses MGMT promoter methylation status, offering less invasive alternatives to traditional brain biopsies.

Higher-Dose Fractionated Reirradiation Shows Promise in Recurrent High-Grade Glioma Treatment

3 months ago

• A Johns Hopkins study of 230 patients demonstrates fractionated reirradiation is safe and effective for recurrent high-grade glioma, with median overall survival reaching 10.2 months from treatment initiation. • Higher reirradiation doses (≥41.4 Gy) proved feasible even for large treatment volumes and tumors near critical brain structures, with minimal neurotoxicity observed in patients. • The study found improved survival outcomes associated with better performance status, longer intervals between radiation sessions, and treatment at first recurrence, supporting this approach as a viable salvage therapy option.

NIH Funding Cuts Under Trump Administration Threaten Early-Stage Biopharma Innovation

3 months ago

• The Trump administration has implemented a $4 billion reduction in NIH overhead funding, cutting indirect cost rates to 15% and disrupting grant review processes for biomedical research. • NIH SBIR and STTR grants for innovative drug development totaled $1.4 billion between 2020-2025, with over 80% supporting crucial preclinical and discovery stage research. • Recent policy changes, including funding freezes and increased scrutiny of grant applications, could significantly impact biotech startups and delay global drug development efforts.

Tiziana Life Sciences Advances Foralumab for MS and SCI Treatment

6 months ago

• Tiziana Life Sciences is expanding its Phase 2 trial of intranasal foralumab for non-active secondary progressive multiple sclerosis (na-SPMS) to include more prestigious U.S. medical centers. • The company announced the discovery of new immune biomarkers in patients with na-SPMS treated with nasal foralumab, enhancing understanding of the drug's mechanism. • Preclinical studies show that nasal anti-CD3 treatment, foralumab, led to notable advancements in motor functions in models with spinal cord injury (SCI). • Tiziana Life Sciences has dosed additional patients in its Expanded Access Program for na-SPMS, with early data showing disease stabilization or improvement.

HIV-Positive Individuals Can Now Receive Kidney and Liver Transplants from HIV-Positive Donors

6 months ago

• The Biden administration has announced new rules allowing people with HIV to receive kidney and liver transplants from HIV-positive donors, expanding access to life-saving organs. • This decision is based on evidence demonstrating the safety and effectiveness of HIV-positive to HIV-positive transplants, eliminating the need for clinical research and IRB approvals. • Studies have shown comparable outcomes between HIV-positive to HIV-positive kidney transplants and transplants from HIV-negative donors to HIV-positive recipients. • The HHS is considering expanding the policy to include heart, lung, pancreas, and other organ transplants, and is seeking public comment on potential revisions.

Induction Chemotherapy Plus Chemoradiotherapy Significantly Improves Survival in Cervical Cancer

7 months ago

• A new study reveals that adding induction chemotherapy (IC) to chemoradiotherapy (CRT) improves survival rates for locally advanced cervical cancer. • The INTERLACE trial showed a 40% reduction in the risk of death and a 35% reduction in cancer recurrence with IC plus CRT. • The treatment regimen involves six weeks of carboplatin and paclitaxel chemotherapy before standard chemoradiotherapy. • Researchers suggest incorporating this approach into clinical guidelines, as it utilizes widely available and affordable drugs.