Regenerative Therapies Library
Every article, presentation, spotlight, and news item we've tagged to Regenerative Therapies.
Showing 121–144 of 297
TDP-43 targeting restores neuronal energy and extends ALS survival
Researchers identified a specific toxic region within TDP-43 protein and developed a small molecule that crosses the blood-brain barrier to target it, demonstrating improved mitochondrial function, reduced neuronal death, and extended survival in ALS models. This represents a mechanistic advance in addressing a protein dysfunction central to multiple neurodegenerative diseases.
Cholesterol Drainage Therapy Reverses Atherosclerosis and Liver Disease
Repair Biotechnologies has developed REP-0004, an mRNA therapy delivered via lipid nanoparticles to the liver, designed to reduce excess intracellular free cholesterol and trigger systemic cholesterol drainage. The drug has received orphan drug designation and aims to enter clinical trials by mid-2027, with preclinical data suggesting rapid regression of atherosclerotic plaque and reversal of metabolic liver disease.
Injection Errors Compound Gray-Market Peptide Risks
Self-administered peptide injections purchased through unregulated gray-market channels carry compounded risks: both contamination from lack of manufacturing oversight and critical injection technique errors that occur despite internet research. A study of 20 participants found that fewer than half completed basic preparation and administration steps correctly, with mistakes spanning sterilization, dosing, needle disposal, and injection site selection.
Evolve Science launches longevity peptide platform
Evolve Science has launched a peptide and longevity platform that differentiates itself through supply chain transparency and batch-level documentation rather than marketing claims. The company sources products exclusively from FDA-registered US compounding pharmacies and requires Certificates of Analysis for every product, addressing a documented gap in quality verification within the longevity market.
Longeveron gains Chinese patent for mesenchymal cell potency assays
Longeveron secured a Chinese patent for mesenchymal stem cell potency assays, extending its intellectual property protection through 2041. This standardization of cell quality assessment is foundational for regulatory approval of cell-based therapies, particularly as the company advances laromestrocel in aging-related frailty and cardiac disease.
Sphingolipid Restoration Targets Motor Neuron Degeneration in ALS
Leal Therapeutics has initiated a Phase 1/2 clinical trial of LTX-002, an intrathecally delivered antisense oligonucleotide designed to restore sphingolipid balance in the central nervous system of ALS patients. The therapeutic targets SPTLC1 to reduce ceramide accumulation implicated in motor neuron degeneration, addressing a mechanistic driver of both genetic and sporadic ALS.
Serum‐Derived Extracellular Vesicles as Biological Indicator of Mobility Resilience in Older Adults
Serum extracellular vesicles carry distinct molecular signatures—small noncoding RNAs and mitochondrial proteins—that differentiate older adults with preserved mobility from those with age-related gait decline. These circulating vesicles appear to mediate brain-muscle communication and may serve as noninvasive biomarkers for identifying individuals at risk of functional decline.
Unnatural Products inks $1.7b cardiovascular disease collab with Novartis
Unnatural Products and Novartis have partnered to develop macrocyclic peptides—molecular structures between small molecules and biologics—targeting protein interactions implicated in cardiovascular disease and age-related conditions. This approach addresses a critical gap in drug development where conventional therapeutics cannot effectively engage disease-driving targets.
TRPML1 Agonist Restores Cellular Cleanup in Aging Brain
Lysoway Therapeutics has dosed the first human participant in a Phase I trial of LW-1017, a small-molecule TRPML1 agonist designed to restore cellular waste-clearance capacity in aging neurons. Rather than targeting accumulated toxic proteins directly, the approach addresses the upstream dysfunction in lysosomal maintenance that permits protein accumulation in the first place.
Nuritas debuts PeptiStrong wellness prototypes at Expo West
Nuritas is presenting PeptiStrong functional food prototypes containing AI-identified bioactive peptides from natural food sources, designed to support metabolic health, muscle maintenance, and stress regulation through familiar consumer formats like yogurt and beverages. The approach represents an effort to translate precision peptide science into accessible mainstream wellness products.
Mesoblast receives IND clearance to proceed to registrational trial
Mesoblast received FDA clearance to advance Ryoncil, a mesenchymal stromal cell therapy, into a registrational trial for Duchenne muscular dystrophy in children aged 5-9. The therapy targets the inflammatory cascade underlying DMD progression, with time-to-stand at 9 months as the primary efficacy endpoint.
NK Cell Therapy Targets Neurodegeneration Beyond Alzheimer's
NkGen Biotech will present Troculeucel, a cryopreserved autologous NK cell therapy, at BIOCHINA 2026, with Phase 2 trials underway for Alzheimer's disease and potential expansion into ALS, frontotemporal dementia, and Parkinson's disease. The therapy represents a cell-based approach to neurodegenerative conditions where conventional pharmacology has shown limited efficacy.
Transdermal Treprostinil Patch Simplifies PAH Management
Corsair Pharma's Phase 1 trial demonstrates that a transdermal patch delivering an inactive precursor of treprostinil can maintain therapeutic drug levels for 24 hours with acceptable skin tolerability. This delivery innovation addresses a critical burden in pulmonary arterial hypertension management: replacing continuous infusion pumps and indwelling catheters with a once-daily application.
XellSmart gets fourth consecutive IND clearances for iPSC cell therapy trials
XellSmart has secured IND clearance for a Phase I/II trial of iPSC-derived neuron progenitor cells targeting multiple system atrophy-Parkinsonian type, marking its fourth consecutive regulatory approval for CNS cell therapies. This represents clinical validation of allogeneic, off-the-shelf regenerative approaches for a rapidly progressive neurodegenerative disease with no disease-modifying treatments.
Sleep-based therapy targets glymphatic clearance in neurodegeneration
AstronauTx has secured exclusive global rights to ATX0926, a sleep-based neurological candidate developed through collaboration with Saniona, targeting sleep architecture and glymphatic clearance mechanisms. The approach represents a shift toward using sleep physiology as a therapeutic lever for neurological disease rather than as a symptom to suppress.
Rubedo Announces Positive Preliminary Results for RLS-1496
Rubedo's RLS-1496, a GPX4 modulator designed to target senescent cells, met primary safety endpoints in a Phase 1 trial and demonstrated dose-dependent clinical improvements in psoriasis, atopic dermatitis, and photoaged skin within four weeks. The compound showed reduced senescent cell burden, decreased inflammatory markers, and increased collagen expression with no serious adverse events.
Startup builds on ‘breakthrough’ discovery to combat ALS
Vesalic has identified a systemic metabolic dysfunction originating outside the brain that drives ALS pathology through circulating exosomes carrying toxic cargo to motor neurons. This reframes ALS as a systemic disease rather than a purely CNS disorder, redirecting therapeutic strategy away from decades of brain-focused approaches with limited success.
Scalable biotech manufacturing targets $14B cell therapy market
Cell therapy manufacturing capacity is becoming a critical bottleneck as the market expands toward $14 billion by 2035. A joint venture between Avaí Bio and Austrianova is scaling production of genetically modified cells engineered to elevate circulating alpha-Klotho, a protein associated with cellular resilience and healthspan.
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.