UNIVERSITY OF SOUTHERN CALIFORNIA

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.

ScaleReady Awards G-Rex Grants to Advance Novel CAR-T Cell Therapy Manufacturing

23 days ago

• ScaleReady has awarded a $200,000 G-Rex Grant to March Biosciences to advance manufacturing of MB-105, a novel CAR-T therapy targeting CD5 for T-cell lymphomas that received FDA orphan drug designation in January 2025. • A2 Biotherapeutics received a $300,000 G-Rex Grant to evaluate the G-CART process, a standardized workflow designed for high-throughput CAR-T manufacturing that can be adapted for commercial scale production. • The G-Rex platform, currently used by over 800 organizations in approximately 50% of CGT clinical trials, aims to reduce complexity, time, and costs in cell therapy manufacturing through a modular and flexible approach.

NeOnc Technologies Strengthens Competitive Edge with 176-Patent Global Portfolio

a month ago

• NeOnc Technologies has built a robust intellectual property portfolio comprising 126 issued patents and 50 pending applications across major pharmaceutical markets worldwide. • The company's patent protections cover its core pipeline of novel oncology and neurology therapeutics, including NEO100 and NEO212, which are currently in Phase II clinical trials under FDA Fast-Track status. • Key patents extend to 2039 and include protection for the company's innovative blood-brain barrier permeabilization technology, positioning NeOnc for long-term market exclusivity.

FDA Grants Rare Pediatric Disease Designation to NEO100 for Aggressive Childhood Brain Cancer

2 months ago

• The FDA has granted Rare Pediatric Disease Designation to NEO100 (perillyl alcohol) for treating pediatric-type diffuse high-grade gliomas, recognizing the urgent need for therapies against this life-threatening brain cancer. • NeOnc Technologies may receive a valuable Priority Review Voucher upon marketing approval, which could expedite the regulatory process and potentially be sold in the dynamic PRV market. • NEO100, administered as a nasal spray, is advancing to a Phase 1b clinical trial to evaluate safety, effectiveness, and optimal dosing in children with aggressive brain tumors.

NeOnc Technologies to List on Nasdaq Global Market with Novel Blood-Brain Barrier Therapeutics

2 months ago

• NeOnc Technologies Holdings, Inc. will begin trading on the Nasdaq Global Market under the ticker symbol "NTHI" on March 26, 2025, following SEC approval of its registration statement. • The clinical-stage biotechnology company is developing central nervous system therapeutics designed to overcome the blood-brain barrier, with its NEO100 and NEO212 candidates currently in Phase II trials. • NeOnc's proprietary NEO drug development platform has demonstrated positive effects against various cancers, particularly malignant gliomas, with both lead candidates advancing under FDA Fast-Track designation.

NeOnc Technologies Partners with CBCC Global Research to Accelerate Brain Cancer Clinical Trials in India

2 months ago

• NeOnc Technologies has formed a strategic partnership with CBCC Global Research to expand its clinical trial network to 30 FDA-compliant research sites across India, focusing on neuro-oncology treatments. • The collaboration will initially focus on the NEO100-01 Phase 2a trial for Glioblastoma (GBM), evaluating an intranasal ultra-purified perillyl alcohol therapy targeting IDH1 mutations in Grade 3 and 4 tumors. • NeOnc aims to complete Phase 2 enrollment this year with results expected 6-8 months later, potentially accelerating the development timeline for novel brain cancer therapies while reaching a more diverse patient population.

Novel Treatment Approaches Show Promise in Brain Cancer: From Aerosolized Drugs to Personalized Vaccines

3 months ago

• Recent developments in brain metastases treatment showcase promising results with targeted therapies like osimertinib, offering improved blood-brain barrier penetration and more focused radiation approaches. • Innovative treatment methods under investigation include NEO100, an aerosolized perillyl alcohol delivered nasally, being tested in meningioma and IDH-mutant glioma patients. • A personalized vaccine approach using patients' tumor tissue is being evaluated in a phase 2b trial for glioblastoma, representing a new frontier in immunotherapy for brain cancer.

American Cancer Society Unveils Strategic Vision: $517M Research Initiative Focuses on Early Detection and Diversity

3 months ago

• The American Cancer Society (ACS) has committed $517 million to research grants, with a significant focus on supporting early-career investigators and promoting diversity in cancer research. • ACS launches targeted programs including VOICES of Black Women study enrolling 100,000 participants and new IMPACT awards addressing prostate cancer disparities in high-risk populations. • The organization emphasizes AI integration in cancer detection and advocates for multicancer detection legislation, demonstrating its commitment to innovation and patient advocacy.

USC Secures $10.8M PCORI Grant to Compare Hip Fracture Surgery Approaches in Elderly Patients

4 months ago

• USC researchers have received a $10.8 million PCORI grant to conduct the FASTER-Hip trial, comparing hip replacement versus internal fixation for minimally displaced femoral neck fractures in older adults. • The study will enroll 600 patients across 32 clinical sites in the US, Canada, and Europe, focusing on patient-centered outcomes including independence and mobility rather than traditional surgical metrics. • Led by Dr. Joseph Patterson, the trial uniquely incorporates input from patients, caregivers, and healthcare professionals to ensure research outcomes align with patient priorities and real-world needs.

Landmark Prevention Trial Launches to Stop Alzheimer's Before Symptoms Begin in Young Adults

4 months ago

• WashU Medicine initiates groundbreaking international trial testing Eli Lilly's remternetug in young adults as young as 18, targeting Alzheimer's prevention up to 25 years before expected symptom onset. • The Primary Prevention Trial will enroll 240 participants from families with genetic mutations, focusing on removing or preventing amyloid beta plaques before cognitive symptoms develop. • The $130 million study represents a collaborative effort between academic institutions, pharmaceutical industry, and foundations, with support from NIH, Alzheimer's Association, and private donors.

Imperative Care's Zoom System Receives FDA Clearance for Stroke Thrombectomy

4 months ago

• Imperative Care's Zoom System, a comprehensive stroke thrombectomy system, has received FDA 510(k) clearance, expanding its portfolio of aspiration catheters. • The Zoom System includes large-bore 0.088-inch catheters indicated for both access and aspiration, offering versatility for rapid and effective stroke procedures. • Clinical trial data demonstrated a median time of 19 minutes from groin puncture to successful reperfusion (mTICI ≥ 2B), the fastest among referenced trials. • The Imperative Trial also showed a low rate of symptomatic intracranial hemorrhage (0.9%) and vessel injury (0.5%), highlighting the system's safety profile.

J&J Seeks FDA Approval for TAR-200 in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

4 months ago

• Johnson & Johnson has initiated a New Drug Application (NDA) submission to the FDA for TAR-200. • TAR-200 is intended for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC). • Phase 2b SunRISe-1 study data showed an 83.5% complete response rate with TAR-200 monotherapy. • The FDA is reviewing the application under the Real-Time Oncology Review (RTOR) program to expedite potential approval.

Study Reveals High Public Willingness to Fund DMD Treatment Through Taxes Despite No Personal Benefit

4 months ago

• A new study finds US adults are willing to pay an average of $80 annually in additional taxes to fund novel Duchenne muscular dystrophy treatments for others, demonstrating significant altruistic value. • The research, focused on individuals not planning to have children, shows strong public support for funding rare pediatric disease treatments even without direct personal benefit. • When unadjusted for decision weights, respondents indicated willingness to pay up to $799 annually, highlighting substantial societal empathy for children with DMD.

NeOnc Technologies' Nasal Spray Shows Promise in Glioblastoma Treatment

4 months ago

• NeOnc Technologies is developing a nasal spray drug delivery system to bypass the blood-brain barrier for glioblastoma treatment. • The innovative approach utilizes cranial nerves to deliver cancer drugs directly to the brain, improving drug penetration. • Early clinical trial results show promising signs of prolonged life and tumor regression in some patients with terminal brain cancer. • The FDA has granted Fast Track designation to NeOnc, expediting the review process for this potential breakthrough therapy.

Semaglutide Shows Promise in Reducing Alcohol Consumption and Cravings

6 months ago

• A new study reveals that semaglutide, known as Ozempic and Wegovy, significantly reduces alcohol cravings and consumption in individuals with alcohol use disorder. • Participants on semaglutide experienced fewer heavy drinking days and decreased alcohol intake during lab tests compared to those on a placebo, indicating potential therapeutic benefits. • The research suggests semaglutide's effects on alcohol cravings may surpass existing treatments, offering a promising avenue for addressing the unmet needs in alcohol use disorder. • Further studies are warranted to explore semaglutide's long-term efficacy, optimal dosages, and safety, particularly for individuals without obesity or diabetes.