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HHS Unveils AI Initiatives to Accelerate U.S. Clinical Trials

HHS Unveils AI Initiatives to Accelerate U.S. Clinical Trials

The U.S. Department of Health and Human Services (HHS) has announced a new program and three complementary projects aimed at significantly speeding up clinical trials through the integration of artificial intelligence (AI), advanced computational models, and real-time analysis. The core initiative, named SURPASS (Simulation-augmented, Real-time Platform Adaptive Seamless Trials), seeks to develop AI-powered platforms that will continuously and adaptively support clinical trial designs. This program includes the creation of a phaseless design engine to simulate trials for faster results with fewer patients, a continuous inference engine for real-time data analysis and trial adaptations, and an agentic AI operations layer to automate startup activities and data collection. These efforts are part of a broader strategy by HHS to transform U.S. clinical trials, building on previous initiatives like Operation TrialBlazer and the FDA's pilot program using AI for real-time clinical trial data monitoring. The goal ...

Eli Lilly's Jaypirca Approved for First-Line Treatment of CLL/SLL in U.S.

Eli Lilly's Jaypirca Approved for First-Line Treatment of CLL/SLL in U.S.

Eli Lilly's Jaypirca Approved for First-Line Treatment of CLL/SLL in U.S.

CRISPR Therapeutics Advances Gene-Edited Therapies for Hemoglobinopathies, Autoimmune Diseases, and Oncology

CRISPR Therapeutics Advances Gene-Edited Therapies for Hemoglobinopathies, Autoimmune Diseases, and Oncology

CRISPR Therapeutics AG is actively developing a diverse portfolio of gene-based medicines utilizing its CRISPR/Cas9 platform. The company's lead product candidate, CASGEVY, is an ex vivo CRISPR/Cas9 gene-edited cell therapy approved for treating transfusion-dependent beta-thalassemia and severe sickle cell disease (SCD). This therapy involves editing a patient's hematopoietic stem and progenitor cells to produce high levels of fetal hemoglobin. Beyond hemoglobinopathies, CRISPR Therapeutics is advancing CAR T cell therapies, including CTX112 and CTX131, which target CD19 and CD70 respectively, for oncology and autoimmune indications. The company is also exploring in vivo gene editing with CTX310 and CTX320 to address cardiovascular disease by disrupting specific protein targets. Additionally, CTX211 is an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate under development for type 1 diabetes. CRISPR Therapeutics maintains strategic partnerships, notably with Vertex Pharmaceuticals Inc...

CRISPR Gene Therapies Advance Towards Direct In-Body Genome Editing for Inherited Retinal Disease

CRISPR Gene Therapies Advance Towards Direct In-Body Genome Editing for Inherited Retinal Disease

CRISPR gene editing therapies are progressing from ex vivo (outside the body) modifications to direct in-vivo (inside the body) genome editing. A significant development in this area is the BRILLIANCE clinical trial, which in 2020 treated its first patient with EDIT-101, an experimental CRISPR/Cas9 therapy for Leber congenital amaurosis type 10 (LCA10). LCA10 is a rare inherited retinal disorder caused by mutations in the CEP290 gene. The treatment involves a subretinal injection designed to deliver the editing machinery directly to retinal cells. The eye is considered an ideal environment for such research due to its contained tissue, allowing for localized treatment delivery. While this represents a major step forward, researchers emphasize that these results should not be mistaken for an established human clinical therapy. The field is also exploring in-vivo approaches for other tissues, such as the liver, using lipid nanoparticles for systemic administration, with some experimental programs already in ...

Soda Steel Gummies Marketed for Male Vitality Lack Clinical Trial Evidence

Soda Steel Gummies Marketed for Male Vitality Lack Clinical Trial Evidence

Soda Steel Gummies Marketed for Male Vitality Lack Clinical Trial Evidence

Enable Biosciences Advances Early Type 1 Diabetes Detection with Predictive Tools

Enable Biosciences Advances Early Type 1 Diabetes Detection with Predictive Tools

Enable Biosciences, a San Francisco-based biotechnology company, has developed predictive medicine tools to identify individuals eligible for preventative therapy for type 1 diabetes (T1D). These tools utilize advanced antibody testing to detect autoantibody biomarkers that appear years before clinical symptoms of T1D manifest. According to Peter Robinson, Co-Founder and Chief Operating Officer of Enable Biosciences, their test is highly effective at predicting who will develop T1D. This early detection is crucial because a drug therapy, approved in November 2022, can delay the onset of T1D, but it must be administered before clinical symptoms or metabolic indications appear. The company's Antibody Detection by Agglutination-PCR (ADAP) technology screens for three major T1D-associated autoantibodies: GAD65, IA-2, and Insulin autoantibodies. While the test is a laboratory-developed test and has not been cleared or approved by the U.S. Food and Drug Administration (FDA), it is offered through a CLIA-certifie...

Regeneron Expands Global Immunology Alliance with Sanofi, Securing $1 Billion Upfront

Regeneron Expands Global Immunology Alliance with Sanofi, Securing $1 Billion Upfront

Regeneron Expands Global Immunology Alliance with Sanofi, Securing $1 Billion Upfront

SereNeuro Therapeutics Secures Pre-Seed Financing to Advance Non-Opioid Pain Therapies

SereNeuro Therapeutics Secures Pre-Seed Financing to Advance Non-Opioid Pain Therapies

SereNeuro Therapeutics Secures Pre-Seed Financing to Advance Non-Opioid Pain Therapies

FDA Launches Expedited IND Pilot Program and Updates Non-Animal Testing Regulations

FDA Launches Expedited IND Pilot Program and Updates Non-Animal Testing Regulations

The U.S. Food and Drug Administration (FDA) has initiated an Expedited Investigational New Drug (IND) Pilot program, now accepting applications. This program aims to accelerate the drug development process by pairing drug sponsors with Qualified Research Institutions (QRIs) to support IND application preparation. The pilot allows for a rolling review of individual IND components during the pre-IND phase, rather than requiring a complete package upfront, with the goal of reducing clinical holds and shortening the drug-to-first-dose timeline. Applications for this pilot are due by October 30, and the FDA anticipates selecting 8–10 sponsor–QRI pairs for the initial cohort. Concurrently, the FDA has issued a direct final rule replacing 'animal tests' with 'nonclinical tests' in its drug and biologics regulations, explicitly allowing non-animal methods like human cell cultures, organs-on-chips, and computer models for pre-human safety assessment. This change does not eliminate animal studies but clarifies that ...

Orano Med Expands Lead-212 Production with New U.S. Facility for Cancer Therapies

Orano Med Expands Lead-212 Production with New U.S. Facility for Cancer Therapies

Orano Med, a clinical-stage biotechnology company and subsidiary of the Orano group, is expanding its global manufacturing capabilities for lead-212-based targeted alpha therapies (TATs) for cancer treatment. Following the inauguration of its ATLab Valenciennes in France, which is Europe's first industrial-scale pharmaceutical facility dedicated to producing these radioligand therapies, Orano Med is also commissioning a similar production site in Indianapolis, in the U.S. This U.S. facility is designed to serve the North American market, mirroring the European site's goal of large-scale production to meet patient needs. The company's TATs utilize lead-212 (212Pb), an alpha-emitting radioisotope, as a potent therapeutic payload against cancer cells. Orano Med's strategy involves developing several 212Pb-based radioligand therapies combined with various targeting agents, with four drug candidates currently in clinical development and a broad preclinical pipeline. The logistical demands for these treatments a...

Nearly 750K eye drops recalled over sterility concerns. See list

Nearly 750K eye drops recalled over sterility concerns. See list

The recall covers nine products, including some sold at Walgreens, and 747,844 units in total.

Cleveland Clinic Weston Hospital Deploys First-in-Florida Chemo Compounding Robot to Enhance Safety and Efficiency

Cleveland Clinic Weston Hospital Deploys First-in-Florida Chemo Compounding Robot to Enhance Safety and Efficiency

Cleveland Clinic Weston Hospital in Florida has become the first hospital in the state to implement the Loccioni APOTECAchemo robotic compounding system. This advanced technology is designed to automate the sterile preparation of hazardous oncology medications, including chemotherapy. The robot handles critical steps such as drug and diluent weighing, vial handling, reconstitution of powdered medications, dose withdrawal, and final container preparation. A key feature of the system is its use of gravimetric verification, which weighs ingredients throughout the preparation process to ensure accurate medication and diluent amounts. This provides an additional layer of safety beyond visual inspection, aiming to prevent underdosing or overdosing. The system also standardizes chemotherapy compounding, ensuring consistency and precision in a high-volume oncology setting through automated checks, barcode verification, and electronic documentation. Dr. Zeina Nahleh, Vice Chief of the Cancer Institute and Regional ...