Regenerative Therapies Library
Every article, presentation, spotlight, and news item we've tagged to Regenerative Therapies.
Showing 145–168 of 310
Edited islet cells persist for a year without immunosuppression
Sana Biotechnology's hypoimmune islet cell transplants achieved one-year persistence and function in a Type 1 diabetes patient without systemic immunosuppression, validating a cell-engineering approach that modifies transplanted cells rather than suppressing the immune system. This durability milestone suggests scalable, off-the-shelf cell therapies may be feasible without the toxicity burden of traditional immunosuppression.
Mitochondrial Therapy FORZINITY Gains FDA Approval for Barth Syndrome
Stealth BioTherapeutics rebranded as Mighty Therapeutics following FDA accelerated approval of FORZINITY (elamipretide), the first FDA-approved therapy directly targeting mitochondrial function for Barth syndrome. The approval represents a significant milestone in mitochondrial medicine, with the company pursuing expanded indications in younger patients and age-related macular degeneration.
Cell therapy advances cardiac recovery in Japanese patients
BioCardia's CardiAMP cell therapy for ischemic heart failure has received regulatory alignment from Japan's PMDA, with an estimated 20,000 eligible patients among Japan's 300,000 with reduced ejection fraction. The pathway forward requires demonstration of patient eligibility criteria and post-marketing surveillance protocols before commercial authorization.
Protac market set for rapid growth through 2034 amid targeted therapy demand
PROTACs—molecules that selectively degrade disease-causing proteins via the cell's natural ubiquitin-proteasome system—represent a mechanistic departure from traditional inhibitor-based therapies. The market is forecast to expand significantly through 2034, driven by 90 protein-degradation leads in development and pipeline expansion across oncology, neurodegenerative, and inflammatory indications.
Mesoblast receives IND clearance for registrational trial of Ryoncil
Mesoblast has received FDA clearance to conduct a registrational trial of Ryoncil, an allogeneic mesenchymal stem cell therapy, in pediatric Duchenne muscular dystrophy patients aged 5-9 years. The trial will assess whether the therapy's anti-inflammatory properties can preserve muscle function and slow disease progression in a population with limited treatment options.
Epigenetic PCSK9 Silencing: Single-Dose LDL Control
Scribe Therapeutics has received regulatory approval to begin human testing of STX-1150, an epigenetic therapy designed to silence PCSK9 and lower LDL cholesterol through a single mRNA-based treatment delivered via lipid nanoparticle to the liver. This represents the first clinical evaluation of a reversible epigenetic silencing approach to cholesterol management, offering potential for sustained LDL reduction without permanent genomic alteration.
Scarlet raises $4m as lab-grown blood matches donor cell survival
Scarlet Therapeutics has demonstrated that laboratory-grown red blood cells survive in circulation as long as donor-derived cells, validating a platform for scalable cell manufacturing and therapeutic delivery. This represents a critical proof-of-concept for replacing donor-dependent blood supply with engineered cells capable of both oxygen transport and targeted therapeutic function.
Urinary detection of therapy-induced senescence and fibrosis using an injectable albumin-based nanoprobe
Researchers developed an injectable nanoprobe that detects cellular senescence through urine analysis, using MMP-7 enzyme activity as a measurable marker. This enables non-invasive, real-time monitoring of treatment response in lung cancer and pulmonary disease, establishing a quantifiable method to track senescence burden during therapy.
Intracellular cholesterol removal opens cardiovascular repair pathway
Repair Biotechnologies has secured FDA Rare Disease Evidence Principles (RDEP) eligibility for REP-0003, an mRNA therapy targeting intracellular free cholesterol accumulation in homozygous familial hypercholesterolemia. This regulatory milestone enables a more tractable clinical pathway for a therapy designed to remove established atherosclerotic damage rather than simply manage lipid levels—a distinction with implications extending beyond inherited cholesterol disorders to broader cardiovascular aging.
Cellular retinoic acid-binding protein 1, CRABP1, in thyroid gland aging
Cellular retinoic acid-binding protein 1 (CRABP1) regulates thyroid aging through vitamin A metabolism and retinoic acid signaling. Dysfunction in this protein correlates with thyroid senescence, positioning CRABP1 as a potential biomarker and intervention target for age-related thyroid decline.
Perivascular Fat Control Restores Vessel Relaxation
A BET inhibitor (RVX-208) that suppresses stress-response gene transcription in perivascular fat restores blood vessel relaxation and reduces inflammatory markers in patients with obesity and hypertension. This approach targets the signaling interface between fat and vessel tissue rather than individual inflammatory molecules, demonstrating that perivascular adipose tissue dysfunction is a primary driver of endothelial impairment in cardiometabolic disease.
Clene reports full year 2025 results with ALS drug progress
Clene Inc. advanced CNM‑Au8, an investigational therapy targeting mitochondrial dysfunction in neurodegenerative diseases, with biomarker data showing statistically significant reductions in neurodegeneration markers linked to improved survival. The company narrowed its net loss to $26.2 million in 2025 and secured over $28 million in funding to support regulatory filings through 2027.
Vasa Therapeutics targets 2026 clinical entry for peripheral artery disease therapy
Vasa Therapeutics is advancing VS-214, an apelin peptide analog designed to promote new blood vessel formation and improve blood flow in peripheral artery disease patients, toward first-in-human trials in 2026. PAD affects 10–12 million Americans and causes approximately 400 non-traumatic amputations daily, representing a significant clinical need for pharmacological intervention.
Coultreon raises $125m for immune resilience
Coultreon Biopharma raised $125 million to advance COL-5671, an oral SIK3 inhibitor designed to restore immune balance rather than blunt inflammatory suppression in autoimmune disease. The approach targets a mechanism relevant to chronic immune dysregulation associated with aging, positioning the therapy as a potential bridge between disease treatment and healthspan optimization.
Mayo Clinic and Sana partner to advance Type 1 diabetes cell therapy
Mayo Clinic and Sana Biotechnology are collaborating to develop SC451, a hypoimmune-modified pancreatic islet cell therapy designed to restore glucose control in type 1 diabetes without requiring ongoing insulin or immunosuppression. The partnership establishes protocols for clinical delivery, patient selection, and monitoring to support Phase 1 trials beginning this year.
Restore Hyper Wellness kicks off NAD Month to educate on IV, IM therapies
Restore Hyper Wellness is promoting NAD supplementation through IV and intramuscular delivery formats, capitalizing on significant year-over-year increases in consumer search interest for NAD-related therapies. The initiative positions NAD precursors and direct NAD administration as tools for cellular energy optimization, though clinical evidence supporting broad longevity claims remains limited.
NAD+ webinar set to examine delivery dilemma
NAD+ delivery methods—pills, injections, and pens—are receiving increased clinical scrutiny as the molecule transitions from niche longevity therapy to mainstream wellness category. The effectiveness of NAD+ depends less on the molecule itself than on bioavailability, formulation quality, and whether patients maintain consistent use.
Senescent cells drive aging inflammation
Cellular senescence—the accumulation of non-dividing cells that secrete inflammatory factors—drives age-related decline in multiple organ systems. Understanding how senescent cells compromise tissue function and identifying interventions that clear or manage their activity offers a direct path to extending both healthspan and lifespan.
Niagen launches telehealth NAD+ injection platform
Niagen Bioscience launched a prescription-only telehealth platform delivering subcutaneous NAD+ injections at home, shifting nicotinamide riboside from over-the-counter supplement to clinician-directed pharmaceutical intervention. The model combines telehealth access with clinical oversight and compounded pharmaceutical-grade dosing, though the clinical evidence supporting NAD+ supplementation for meaningful longevity outcomes remains incomplete.
Niagen Bioscience secures patent for NR injections
Niagen Bioscience secured a US patent protecting intravenous and injectable formulations of nicotinamide riboside (NR), a NAD+ precursor molecule that declines with age and cellular stress. The patent extends protection through 2044 and positions the company to dominate clinical delivery of NAD+ boosters, a market rapidly expanding across wellness clinics.
Increasing Senolytic Effectiveness by Stressing Mitochondria
Mitochondrial stress emerges as a critical mechanism underlying senolytic effectiveness against senescent cells. This finding suggests that the therapeutic benefit of senolytics depends partly on their capacity to induce cellular energy stress, offering a framework for optimizing drug selection and combination strategies in senescence-targeted interventions.
Klotho unveils AI-powered aging clock
Klotho Neurosciences has developed AI-powered genomics tests that measure biological age through DNA methylation and mRNA analysis of longevity-associated genes. This approach enables more precise stratification in clinical trials for neurodegenerative diseases, reducing confounding variables that arise when chronological and biological age diverge.
Senolytic Treatment With Fisetin Reverses Age‐Related Endothelial Dysfunction Partially Mediated by SASP Factor CXCL12
Fisetin, a plant-derived senolytic, reverses age-related endothelial dysfunction in aging mice by eliminating senescent endothelial cells and reducing the SASP factor CXCL12, which drives vascular dysfunction through oxidative stress and impaired nitric oxide production. This identifies a mechanistic pathway linking cellular senescence to cardiovascular aging and demonstrates functional recovery through targeted senolytic intervention.
Senescent Cells Block Cellular Cleanup in Aging
Cellular senescence and autophagy dysfunction accumulate with age, driving multiple organ system decline. Understanding how senescent cells disrupt normal repair mechanisms reveals intervention points for extending healthspan independent of chronological age.