UNIVERSITY OF CALIFORNIA SAN DIEGO

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.

Mindfulness and CBT Significantly Reduce Chronic Low Back Pain and Opioid Use in Landmark Clinical Trial

a month ago

• A large clinical trial published in JAMA Network Open demonstrates that both mindfulness meditation and cognitive-behavioral therapy significantly reduced pain in patients with chronic low back pain severe enough to require opioid treatment. • Participants who received eight weeks of therapist-led group sessions reported lasting improvements in pain levels, physical function, and quality of life, with many spontaneously reducing their opioid medication use. • Researchers believe these non-pharmacological approaches work by engaging distinct brain mechanisms that directly modify pain perception, offering promising tools for managing various chronic pain conditions.

Novel Drug Lorundrostat Shows Significant Promise for Uncontrolled Hypertension in Phase 2b Trial

2 months ago

• Cleveland Clinic-led clinical trial demonstrates lorundrostat, an aldosterone synthase inhibitor, reduced systolic blood pressure by 8 points more than placebo in patients with uncontrolled hypertension. • The 12-week Advance-HTN trial included 285 participants with 53% being Black Americans, a population that carries disproportionately high risk for hypertension complications. • Lorundrostat represents a new class of blood pressure medications that directly reduces aldosterone production, potentially offering treatment options for the 30-40% of patients whose hypertension remains uncontrolled with current therapies.

CROI 2025 to Showcase Breakthrough Research in HIV, COVID-19, and Viral Infections

3 months ago

• The Conference on Retroviruses and Opportunistic Infections (CROI) 2025 will convene in San Francisco from March 9-12, featuring cutting-edge research on HIV cure strategies, COVID-19 variants, and viral infections. • Late-breaking sessions will present phase III clinical trials of novel antivirals, including ensitrelvir for COVID-19 prevention and doravirine/islatravir for HIV treatment. • The conference will address critical public health initiatives, including HIV self-testing programs, doxycycline post-exposure prophylaxis (doxyPEP), and machine learning applications in HIV care management.

UCSD to Evaluate Nexalin's HALO Device for PTSD and Brain Injury Treatment in Independent Clinical Trial

3 months ago

• University of California, San Diego has received IRB approval to conduct independent clinical trials of Nexalin's HALO Clarity device for treating mild traumatic brain injury and PTSD. • The study will deploy 50 HALO devices utilizing Deep Intracranial Frequency Stimulation technology, offering a non-invasive, drug-free treatment option that can be administered at home. • Nexalin's virtual clinic model incorporates AI-powered monitoring and telemedicine capabilities, enabling remote patient tracking and real-time treatment adjustments throughout the trial.

FDA Updates Viloxazine Label with New Pharmacodynamic and Lactation Data for ADHD Treatment

4 months ago

• The FDA has approved an updated label for viloxazine extended-release capsules (Qelbree) for ADHD, including new pharmacodynamic data. • The updated label provides insights into viloxazine's effects on the serotonin 5-HT2C receptor and norepinephrine transporter inhibition. • New data on lactation indicates low transfer of viloxazine into breast milk, offering crucial information for breastfeeding mothers. • Clinical trial data supports the efficacy of viloxazine in both pediatric and adult ADHD patients, showing significant improvements in ADHD Rating Scale scores.

Lexicon to Host Expert Panel on LX9211, Novel Non-Opioid Treatment for Diabetic Neuropathic Pain

4 months ago

• Lexicon Pharmaceuticals will host a virtual webcast on January 28, 2025, featuring an in-depth discussion of LX9211, their investigational non-opioid treatment for diabetic peripheral neuropathic pain. • The event will showcase presentations from Lexicon's leadership team and include expert perspectives from prominent diabetes specialists, including the 2023 ADA President for Medicine and Science. • The webcast comes ahead of anticipated topline data from the Phase 2b PROGRESS trial, positioning LX9211 as a potential paradigm-shifting treatment for neuropathic pain.

Tecovirimat Fails to Improve Mpox Outcomes in NIH-Sponsored STOMP Trial

5 months ago

• The STOMP trial found that tecovirimat did not significantly reduce lesion resolution time or pain in adults with mild to moderate mpox. • The Data Safety and Monitoring Board recommended halting further enrollment due to the lack of efficacy, leading NIAID to close the study. • Tecovirimat remains available through the CDC's expanded access program for eligible patients with severe mpox or immunocompromised conditions. • Researchers emphasize the importance of randomized clinical trials during outbreaks, despite the disappointing results for tecovirimat.

Advances in Metastatic Prostate Cancer: Novel Treatment Strategies Emerge

6 months ago

• The ARANOTE trial demonstrated that darolutamide plus ADT significantly improves radiographic progression-free survival in mHSPC patients. • PEACE-3 trial results highlighted that combining radium-223 with enzalutamide significantly improved outcomes in mCRPC patients with bone metastases. • SPLASH trial data suggests 177Lu-PNT2002 may offer a new radioligand therapy option for mCRPC patients who have progressed after an ARPI.

Wearable Skin Patch Offers Continuous Blood Pressure Monitoring

6 months ago

• Researchers have developed a wearable ultrasound patch for continuous blood pressure monitoring, offering a potential alternative to traditional cuff measurements. • The patch uses ultrasound waves to track changes in blood vessel diameter, converting these changes into blood pressure values, and has shown accuracy comparable to invasive methods. • Testing across various conditions, including daily activities and clinical settings, demonstrated the patch's reliability in providing accurate blood pressure readings. • The development team plans to refine the device and conduct larger clinical trials to further validate its efficacy and potential for widespread use.

FDA Advisory Committee Rejects Zynquista for Type 1 Diabetes and Chronic Kidney Disease

7 months ago

• The FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted against approving Zynquista (sotagliflozin) for adults with type 1 diabetes and chronic kidney disease. • The committee cited concerns that the benefits of Zynquista do not outweigh the risks in the proposed patient population. • Lexicon Pharmaceuticals expressed disappointment but remains hopeful, emphasizing the unmet need for new treatments in this area. • Some committee members supported sotagliflozin for specific subpopulations of T1D and CKD patients, where benefits may outweigh risks.

Electric Skin Patch Shows Promise in Drug-Free Wound Healing by Reducing Bacterial Infections

7 months ago

• A novel skin patch delivering imperceptible low-level electric current significantly reduced Staphylococcus epidermidis, a common skin bacterium, by nearly tenfold. • The bioelectronic patch, named BLAST, creates an acidic environment conducive to the electric current's antibacterial effect, showing potential for treating skin infections. • Researchers observed that the electric current's effectiveness against S. epidermidis was pH-dependent, requiring an acidic environment to inhibit bacterial growth. • This drug-free approach offers a promising alternative to antibiotics, especially against antibiotic-resistant bacteria, and could revolutionize wound healing and infection control.

Vertex's Non-Opioid Pain Drug VX-548 Achieves Positive Phase 3 Results

a year ago

• Vertex Pharmaceuticals' VX-548 demonstrated statistically significant pain reduction compared to placebo in Phase 3 trials for acute pain following abdominoplasty and bunionectomy. • The drug targets NaV1.8 sodium channels in peripheral nerves, offering a novel non-opioid mechanism for pain relief and potentially avoiding the risk of addiction. • While VX-548 was not superior to hydrocodone/acetaminophen in both trials, it showed a similar safety profile and was generally well-tolerated, with manageable side effects. • Vertex plans to submit a New Drug Application to the FDA by mid-year, aiming to provide a new option for acute pain management with a potentially improved safety profile.

RNA-Targeted Drug Discovery Summit to Address Key Challenges in Small Molecule Development

5 years ago

• Leading biopharmaceutical executives and researchers gather at the 3rd RNA-Targeted Drug Discovery Summit to advance RNA-targeting small molecule therapeutics development and improve patient outcomes. • The summit aims to tackle critical challenges in RNA drug discovery, including RNA structure determination, target selection optimization, and translation into clinical development. • Industry leaders from Scripps Research Institute, Anima Biotech, and Arrakis Therapeutics will share insights on enhancing medicinal chemistry approaches and RNA biology understanding for robust therapeutic development.