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Clinical Trial News

CRISPR-Cas9 Corrects Dystrophin Duplications in DMD Patient Cells

  • Researchers used CRISPR-Cas9 to target intronic regions in the DMD gene in patient cells, aiming to delete duplicated regions.
  • The study confirmed restoration of the DMD open reading frame and rescued dystrophin expression after CRISPR-based deletion.
  • Off-target analysis revealed no significant unintended gene editing at predicted close-match off-target loci, enhancing safety profile.
  • This research presents a potential therapeutic avenue for DMD patients carrying duplications in exons 2-9, where no treatment exists.

PBM Transparency Crisis: Industry Expert Calls for Reform to Improve Drug Access and Pricing

  • PBMs significantly impact healthcare costs and medication access through their control of drug formularies and pricing negotiations, affecting patient treatment options and affordability.
  • Current vertical integration in the PBM industry, where companies own retail pharmacies and specialty fulfillment services, creates concerning conflicts of interest and potential price manipulation.
  • Industry expert Fred Barton emphasizes the urgent need for increased data transparency, reduction of anti-competitive practices, and reform of PBM operations to ensure fair drug pricing.

CD137-Targeted Therapies Show Promise in Cancer Immunotherapy

  • CD137-targeted therapies are emerging as a promising approach in cancer immunotherapy due to their ability to stimulate and expand cytotoxic T cells, enhancing tumor cell killing.
  • Several pharmaceutical companies, including BioNTech and Genmab, are actively developing CD137-targeted therapies, with multiple candidates in clinical trials, primarily in Phase 2.
  • Combination therapies involving CD137 agonists with checkpoint inhibitors or chemotherapy show synergistic benefits, potentially improving outcomes for difficult-to-treat malignancies.
  • The market for CD137-targeted therapies is expected to grow significantly, driven by the demand for effective treatments and the potential to address limitations of existing immunotherapies.

Analytic Models Evaluated for Individually Randomized Group Treatment Trials

  • Researchers found that in individually randomized group treatment (IRGT) trials, the analytic model should account for correlations in outcome measures when participants receive an intervention from the same source.
  • The study addressed a knowledge gap by examining analytic models for IRGT trials with complex clustering arising from participants interacting with multiple agents or single agents in nested and crossed designs.
  • The study revealed substantial inflation in the type I error rate in studies with nested designs when the analytic model did not account for participants interacting with multiple agents.
  • The NIH Collaboratory’s Biostatistics and Study Design Core conducted a simulation study to examine the performance of a variety of analytic models for IRGT trials.

Ryvu Therapeutics Advances RVU305 PRMT5 Inhibitor to Preclinical Development; Reports Positive Financials and Clinical Trial Updates

  • Ryvu Therapeutics is advancing RVU305, a PRMT5 inhibitor, into preclinical development with IND/CTA filing targeted for H2 2025.
  • The company's RVU120 Phase II studies are progressing, with initial data expected at the ASH 2024 conference in December.
  • Ryvu's cash position reached USD 65.3 million as of September 5, 2024, providing a financial runway through Q1 2026.
  • Total operating revenues in H1 2024 increased to USD 12.1 million, a USD 4.2 million increase compared to H1 2023.

Endometrial Cancer Clinical Trial Landscape Shows Increasing Global Activity

  • A new report indicates a rise in endometrial cancer clinical trials globally, with the Asia-Pacific region experiencing the highest growth rate since 2014.
  • The United States and China lead in venture capital funding for endometrial cancer research, investing $2129 million and $894 million, respectively, between 2019 and 2023.
  • Clinical trials in North America and Asia-Pacific are focused on early to mid-phase studies, while Europe concentrates on later-stage trials for endometrial cancer.
  • Immunotherapies targeting Programmed Cell Death 1 Ligand 1 and Programmed Cell Death Protein 1 are prominent in Phase III trials for endometrial cancer.

Epilepsy Clinical Trial Landscape Shows Asia-Pacific Leading Research Activity

  • A recent report analyzes the global epilepsy clinical trial landscape, revealing over 4,000 trials initiated in the past 5 years, highlighting significant research efforts.
  • The Asia-Pacific region leads in epilepsy clinical trials, accounting for 35% of global activity, driven by shorter recruitment durations and faster rates.
  • Despite advancements, 50% of epilepsy cases have unknown causes, presenting ongoing challenges in treatment and management, spurring research into new therapies.
  • Venture capital funding for epilepsy research has shown a positive trend, with the United States leading investments, followed by Switzerland and China.

Global Liver Cancer Trial Landscape Shows Robust Activity in Asia-Pacific and North America

  • A new report reveals over 1,700 liver cancer trials initiated globally since 2018, highlighting significant investment in improving patient outcomes.
  • Asia-Pacific leads in trial volume, accounting for 55% of studies, followed by North America at 24%, indicating regional strengths in clinical research.
  • The hepatocellular carcinoma (HCC) treatment landscape is evolving, with diverse approaches including surgery, targeted therapies, and immunotherapies.
  • Funding for liver cancer research is substantial, particularly in the United States and China, supporting preclinical and early-phase clinical trials.

Global CLL Trial Landscape Shows Robust Activity, Highlights Regional Disparities

• A new report reveals that over 1,000 CLL clinical trials have been initiated globally since 2019, with North America leading at 37%, followed by Asia-Pacific at 34%. • CLL drug development includes 24 drugs in preclinical stages, 33 in Phase I, 30 in Phase II, and 4 in Phase III, with Bruton Tyrosine Kinase inhibitors dominating Phase III trials. • China led venture capital funding for CLL research from 2019 to 2023 with $1,813 million, closely followed by the United States with $1,805.6 million. • Targeted therapies, immunotherapy (CAR T-cell therapy), and precision medicine advancements have improved CLL treatment outcomes, with companies like AbbVie and BeiGene actively developing new therapies.

BMI Thresholds Impact Mortality Risk in NSCLC Immunotherapy vs. Chemotherapy

  • A study of over 500,000 lung cancer patients reveals that BMI affects mortality risk differently in immunotherapy versus chemotherapy.
  • For NSCLC patients with a BMI under 28, immunotherapy was associated with lower mortality compared to chemotherapy.
  • In patients with a BMI of 28 or higher, chemotherapy showed a continued decrease in mortality risk, while immunotherapy's risk increased.
  • Findings suggest BMI should be considered when selecting treatment for advanced non-small cell lung cancer.

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