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CRISPR Therapeutics Reports Positive Phase 1 Data for Zugo-cel in Autoimmune Diseases

CRISPR Therapeutics Reports Positive Phase 1 Data for Zugo-cel in Autoimmune Diseases

CRISPR Therapeutics has provided an update on zugocabtagene geleucel (zugo-cel), its investigational allogeneic CAR T cell therapy, in a Phase 1 basket trial for refractory rheumatologic autoimmune diseases. The trial includes patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and idiopathic inflammatory myopathy (IIM). As of October 15, 2026, 21 patients have been dosed across these cohorts: four with SLE, eight with SSc, and nine with IIM. Data presented at ACR Convergence 2026 showed promising results, particularly in SSc patients, where 83% (5/6) receiving standard lymphodepletion achieved a clinical response (rCRISS-25) at Month 3, with all responders maintaining their response. Additionally, all nine IIM patients achieved a moderate or major Total Improvement Score (TIS) response. The company reported that Zugo-cel was well tolerated across all 21 patients, with no cytokine release syndrome (CRS) above Grade 2 and no immune effector cell-associated neurotoxicity syndrome (ICA...
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Johnson Johnson Announces Positive Phase 3 Results for ICOTYDE in Psoriatic Arthritis Treatment

Johnson Johnson Announces Positive Phase 3 Results for ICOTYDE in Psoriatic Arthritis Treatment

Johnson & Johnson has announced positive results from a Phase 3 investigational study for its drug ICOTYDE (icotrokinra) in treating biologic-naive adults with active psoriatic arthritis (PsA). The late-breaking data, presented at the American College of Rheumatology (ACR) Convergence 2026, showed that 58% of patients treated with ICOTYDE achieved an ACR20 response at Week 16, compared to 41% receiving a placebo. Furthermore, 29% achieved an ACR50 response. ICOTYDE is described as the first and only targeted oral peptide that precisely blocks the IL-23 receptor, a key driver of inflammation in psoriatic diseases. The study also reported significant improvements in skin involvement, with 57% of patients achieving clear or almost clear skin (Investigator's Global Assessment 0/1) and 37% achieving a Psoriasis Area and Severity Index (PASI) score of 90, compared to placebo groups. Safety findings were consistent with the established safety profile of ICOTYDE, with no new safety signals identified through Week ...
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Gene Therapy Sponsors Urged to Address Manufacturing Considerations Earlier to Avoid Delays

Gene Therapy Sponsors Urged to Address Manufacturing Considerations Earlier to Avoid Delays

Gene Therapy Sponsors Urged to Address Manufacturing Considerations Earlier to Avoid Delays
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FDA Proposes New Guidance to Accelerate Gene Therapies for Rare Diseases

FDA Proposes New Guidance to Accelerate Gene Therapies for Rare Diseases

The U.S. Food and Drug Administration (FDA) is shaping new guidance that could significantly speed up the development of gene therapies for rare diseases. This initiative, influenced by scientists in Philadelphia, aims to allow researchers to test a gene therapy platform against a sample of mutations. If successful, this would enable customization for individual patients without requiring a new clinical trial for each specific mutation. The FDA's new Rare Disease Framework Initiative emphasizes integrating patient perspectives early in the drug development cycle, moving away from traditional late-stage feedback. This framework champions the use of cutting-edge science, novel methods, and innovative clinical trial designs, including externally controlled studies, particularly for ultrarare diseases affecting fewer than 1,000 individuals. Under the Rare Disease Evidence Principles (RDEP) program, treatments for these ultrarare conditions may only need evidence from a single-arm trial, supplemented by strong ...
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Trialtrove Platform Enhances Clinical Trial Intelligence with AI Integration

Trialtrove Platform Enhances Clinical Trial Intelligence with AI Integration

Trialtrove Platform Enhances Clinical Trial Intelligence with AI Integration
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Dr. Drew Weissman's Discoveries in mRNA Technology Lead to Nobel Prize and New Therapeutic Applications

Dr. Drew Weissman's Discoveries in mRNA Technology Lead to Nobel Prize and New Therapeutic Applications

Dr. Drew Weissman, an American physician and immunologist, was awarded the 2023 Nobel Prize in Physiology or Medicine alongside Katalin Karikó for their discoveries concerning RNA modifications that enabled effective mRNA vaccines against COVID-19. Weissman's career began with studies on HIV interactions with immune cells before he collaborated with Karikó in the late 1990s, leading to their seminal 2005 study on modified nucleosides and immune recognition. Their work continued, demonstrating the advantages of incorporating pseudouridine into mRNA for greater protein production and biological stability, and later addressing issues of double-stranded RNA contaminants. Beyond COVID-19, Weissman has contributed to research on mRNA Zika vaccines and a universal influenza vaccine. His work also extends to cell reprogramming, with studies using mRNA to generate temporary CAR T cells for cardiac fibrosis and delivering mRNA to hematopoietic stem cells for conditions like sickle-cell disease. He is a founding scie...
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Onchilles Pharma Reports Promising Early Clinical Data for N17350, a Personalized Cancer Vaccine Approach

Onchilles Pharma Reports Promising Early Clinical Data for N17350, a Personalized Cancer Vaccine Approach

Onchilles Pharma, a clinical-stage biotechnology company, has reported initial clinical findings from its Phase 1/2 trial (OP-NEU-101) of intratumoral N17350 in patients with advanced solid tumors. The data, presented at the 41st Annual Meeting of the Society for Immunotherapy of Cancer (SITC), suggest that N17350 could effectively turn an injected tumor into a personalized cancer vaccine without requiring tumor sequencing or custom manufacturing. N17350 is an engineered therapeutic elastase derived from a cancer-killing enzyme found in human neutrophils. When injected into a tumor, it selectively kills cancer cells and releases the tumor's own antigens, thereby training the immune system to attack cancer elsewhere in the body. In the first dose cohort, three heavily pretreated patients showed no dose-limiting toxicities. Notably, a patient with Merkel cell carcinoma experienced the disappearance of five non-injected lesions after N17350 was injected into a single lesion, indicating systemic immune activat...
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Precision BioSciences Announces Promising PBGENE-HBV Data for Chronic Hepatitis B Viral Cure

Precision BioSciences Announces Promising PBGENE-HBV Data for Chronic Hepatitis B Viral Cure

Precision BioSciences Announces Promising PBGENE-HBV Data for Chronic Hepatitis B Viral Cure
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Moderna Reports Strong Q3 2026 Earnings, Raises Full-Year Guidance, and Advances Oncology Pipeline

Moderna Reports Strong Q3 2026 Earnings, Raises Full-Year Guidance, and Advances Oncology Pipeline

Moderna Reports Strong Q3 2026 Earnings, Raises Full-Year Guidance, and Advances Oncology Pipeline
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Agenus Reports Strong Third Quarter 2026 Results and Advances Phase 3 ROBBIN Study for Colon Cancer

Agenus Reports Strong Third Quarter 2026 Results and Advances Phase 3 ROBBIN Study for Colon Cancer

Agenus Reports Strong Third Quarter 2026 Results and Advances Phase 3 ROBBIN Study for Colon Cancer
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Moderna Reports 29% Revenue Growth to $1.3 Billion in Q3 2026, Raises Full-Year Guidance

Moderna Reports 29% Revenue Growth to $1.3 Billion in Q3 2026, Raises Full-Year Guidance

Moderna Reports 29% Revenue Growth to $1.3 Billion in Q3 2026, Raises Full-Year Guidance
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City of Hope Researchers Develop Novel Microbiome Therapy to Prevent Bloodstream Infections in Cancer Patients

City of Hope Researchers Develop Novel Microbiome Therapy to Prevent Bloodstream Infections in Cancer Patients

Researchers at City of Hope, in collaboration with Memorial Sloan Kettering Cancer Center and Seres Therapeutics, have developed a new microbiome therapy, SER-155, aimed at preventing dangerous bloodstream infections in patients undergoing donor stem cell transplants for blood cancers. This first-of-its-kind therapy delivers 16 carefully selected beneficial bacteria designed to restore the microbiome and combat harmful pathogens. In a multicenter phase 1 clinical trial, SER-155 was found to be well tolerated with no treatment-related side effects. Patients who received the therapy experienced fewer bloodstream infections and lower levels of harmful bacteria associated with infection. The study, published in Nature Medicine, provides early evidence that live biotherapeutics, which are medicines made from beneficial bacteria, can be safely administered to highly vulnerable patients and may help prevent serious infections. This approach represents a significant step towards engineering the microbiome for spec...