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CoRegen's CRG-150, a Gene-Edited Regulatory T-Cell Therapy, Cleared for Phase 1/2a Clinical Trial in Cancer

CoRegen's CRG-150, a Gene-Edited Regulatory T-Cell Therapy, Cleared for Phase 1/2a Clinical Trial in Cancer

CoRegen has received U.S. Food and Drug Administration (FDA) clearance for its Investigational New Drug (IND) application for CRG-150, a novel gene-edited regulatory T-cell (Treg) therapy. This clearance allows CoRegen to proceed with a first-in-human Phase 1/2a clinical trial (NCT07730125) for patients with metastatic triple-negative breast cancer, metastatic hormone receptor-positive/HER2-negative breast cancer, or metastatic prostate cancer. The therapy involves collecting a patient's own Tregs through apheresis, then using CRISPR-Cas9 gene editing to disrupt the NCOA3 gene, which produces the SRC-3 protein. This reprogramming aims to alter the Tregs' function from suppressing immune responses to actively promoting an anti-tumor immune environment. Unlike many existing cell therapies, CRG-150 does not require lymphodepleting chemotherapy prior to infusion, and the edited cells are intended to reshape the tumor microenvironment and recruit the patient's existing effector immune cells to fight the cancer.

Digital Twins Revolutionize Drug Development by Enhancing Simulation and Predictive Capabilities

Digital Twins Revolutionize Drug Development by Enhancing Simulation and Predictive Capabilities

Digital Twins Revolutionize Drug Development by Enhancing Simulation and Predictive Capabilities

FDA Approves MIMRYLO for Adults with Polycythemia Vera, Addressing Unmet Need

FDA Approves MIMRYLO for Adults with Polycythemia Vera, Addressing Unmet Need

The U.S. Food and Drug Administration (FDA) has approved MIMRYLO (rusfertide) for the treatment of adults diagnosed with polycythemia vera (PV) and erythrocytosis. This marks a significant development as MIMRYLO is the first approved therapy that functions by mimicking hepcidin, a hormone crucial for regulating iron availability and the production of red blood cells. The approval is largely based on compelling data from the Phase 3 VERIFY trial, which demonstrated substantially improved hematocrit control and a notable reduction in the need for phlebotomy procedures when compared to a placebo combined with standard care. Polycythemia vera is a chronic blood cancer characterized by an overproduction of red blood cells, leading to increased blood viscosity and a higher risk of serious cardiovascular complications such as thrombosis and stroke. The primary goal in treating PV is to maintain hematocrit levels below 45%, a challenge for many patients who often require frequent phlebotomies despite receiving sta...

Agenus Publishes Peer-Reviewed Data on BOT+BAL's Efficacy in Metastatic Colorectal Cancer

Agenus Publishes Peer-Reviewed Data on BOT+BAL's Efficacy in Metastatic Colorectal Cancer

Agenus Inc. has announced the peer-reviewed publication of mature Phase 1b results for its combination therapy, botensilimab (BOT) and balstilimab (BAL), in treating microsatellite-stable (MSS) metastatic colorectal cancer (mCRC) without active liver metastases. The study, published in Clinical Cancer Research, involved 123 heavily pre-treated patients who had received a median of three prior lines of therapy. The results showed a median overall survival of 21.2 months and a three-year overall survival rate of 33%. This is a significant improvement compared to available later-line standard treatments, which typically report median overall survival of 10-14 months in this patient group. The therapy demonstrated durable clinical activity even in tumors lacking conventional markers of checkpoint sensitivity, such as low tumor mutational burden (TMB) and no detectable PD-L1 expression, suggesting BOT's Fc-enhanced immune priming mechanism is effective in immunologically 'cold' tumors.

Research Links Nighttime Micro-Awakenings to Higher Genetic Alzheimer's Risk in Middle-Aged Adults

Research Links Nighttime Micro-Awakenings to Higher Genetic Alzheimer's Risk in Middle-Aged Adults

Researchers at ULiège, supported by the Stop Alzheimer's Foundation, have identified a potential early indicator for Alzheimer's disease: frequent nighttime micro-awakenings. A study involving over 500 healthy individuals revealed that middle-aged participants with more frequent micro-awakenings during sleep also exhibited a higher genetic risk for developing Alzheimer's disease. This association was not observed in younger adults. Micro-awakenings are brief bursts of brain activity that interrupt the sleep cycle without fully waking an individual. The findings, published in the journal Sleep, suggest that sleep measurements could eventually aid in identifying individuals more vulnerable to Alzheimer's before the onset of noticeable symptoms. The study also explored the locus coeruleus, a tiny brain region crucial for regulating wakefulness and sleep, noting that it is among the earliest areas where abnormal protein deposits linked to Alzheimer's can appear.

Novo Nordisk Explores Implantable Wegovy to Enhance GLP-1 Drug Delivery

Novo Nordisk Explores Implantable Wegovy to Enhance GLP-1 Drug Delivery

Novo Nordisk Explores Implantable Wegovy to Enhance GLP-1 Drug Delivery

Dr. Steven Austad Discusses Longevity Research and Billionaire Investments

Dr. Steven Austad Discusses Longevity Research and Billionaire Investments

Dr. Steven Austad, a prominent researcher in evolutionary gerontology, discussed the current state of longevity research, the involvement of billionaires, and the challenges of translating animal studies to human applications. He noted that while billionaires are investing heavily in longevity companies, they are generally waiting for convincing evidence before personally taking experimental drugs like rapamycin. Austad emphasized the need for interventions to be accessible to the general public, not just the wealthy. He expressed skepticism about many anti-aging supplement claims, including resveratrol and NAD enhancers, due to a lack of convincing efficacy and clinical benefit. The discussion also covered promising drug candidates like GLP-1 drugs and SGLT2 inhibitors, highlighting the accelerated pace at which plausible interventions are moving towards human studies. Austad maintains a wager that someone alive today will reach 150 years, attributing this potential to a combination of drugs and optimized...

CRISPR-Based Gene Therapy Shows Promise in Sustaining Lower Lipid Levels for a Year

CRISPR-Based Gene Therapy Shows Promise in Sustaining Lower Lipid Levels for a Year

Researchers have reported promising results from a small study involving a one-time CRISPR-based gene therapy shot that helped individuals with elevated cholesterol and triglyceride levels maintain significantly lower lipid levels for at least a year. Mel Mann, a patient advocate, highlighted this development, noting that the therapy represents a 'different model' compared to traditional treatments. The study, though small and involving severe cases, demonstrated that the highest dose of the CRISPR shot reduced LDL cholesterol and triglycerides by approximately half, with the effects sustained over the year. This suggests a potential for a durable therapeutic intervention for lipid management through gene editing.

Oryon Cell Therapies Appoints Kenny Choi, Ph.D. as Chief Technical Officer to Lead Manufacturing Strategy

Oryon Cell Therapies Appoints Kenny Choi, Ph.D. as Chief Technical Officer to Lead Manufacturing Strategy

Oryon Cell Therapies Appoints Kenny Choi, Ph.D. as Chief Technical Officer to Lead Manufacturing Strategy

Compass Pathways Seeks Director for U.S. Site of Care to Advance Psilocybin Therapy for PTSD

Compass Pathways Seeks Director for U.S. Site of Care to Advance Psilocybin Therapy for PTSD

Compass Pathways, a biotechnology company headquartered in London with a U.S. office in New Jersey, is actively developing COMP360 psilocybin, an investigational treatment for Post-traumatic Stress Disorder (PTSD) and treatment-resistant depression (TRD). COMP360 has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for TRD. The company is currently executing a late-stage trial for COMP360 in PTSD and is seeking a Director, National Site of Care in the United States. This role is crucial for establishing strategic relationships with organized care groups, health systems, and treatment centers that will serve as early adopters of COMP360 therapy. The Director will lead a team responsible for site-of-care execution, ensuring consistent account planning, customer engagement, and launch readiness activities across the U.S.

Takeda's MIMRYLO™ (rusfertide) Approved by U.S. FDA for Polycythemia Vera Erythrocytosis

Takeda's MIMRYLO™ (rusfertide) Approved by U.S. FDA for Polycythemia Vera Erythrocytosis

Takeda has announced that the U.S. Food and Drug Administration (FDA) has approved MIMRYLO™ (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV), a blood cancer. MIMRYLO is a first-in-class hepcidin mimetic designed to regulate iron distribution and red blood cell overproduction, aiming to control hematocrit levels. Maintaining hematocrit below 45% is the primary treatment goal for PV patients to prevent life-threatening thrombotic events. The approval is supported by data from the global randomized Phase 3 VERIFY study, which included 293 PV patients. The study showed that MIMRYLO, combined with standard of care, resulted in a higher response rate, reduced phlebotomy needs, and improved fatigue compared to placebo. MIMRYLO is administered once weekly via subcutaneous injection and was generally well-tolerated in trials, with common adverse events being injection site reactions and anemia.

Neurovalens Receives FDA De Novo Approval for Modius Spero PTSD Treatment

Neurovalens Receives FDA De Novo Approval for Modius Spero PTSD Treatment

Neurovalens, a biotechnology company specializing in non-invasive neuro-technology, has announced the publication of peer-reviewed research in Nature Mental Health detailing the effectiveness of its Modius Spero device for treating symptoms associated with Post-traumatic Stress Disorder (PTSD). The device, which uses low-level electrical pulses to stimulate areas of the brain regulating stress response, recently received De Novo approval from the U.S. Food & Drug Administration (FDA). This approval allows Modius Spero to be prescribed to veterans through the U.S. Department of Veterans Affairs (VA). The clinical trial, conducted by the University of California, San Diego, involved 383 U.S. adults with PTSD and demonstrated statistically and clinically significant reductions in both PTSD symptom severity and insomnia. This marks a significant development in providing a non-invasive, at-home treatment option for individuals living with PTSD.