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Homology Medicines Announces Optimized, In Vivo Gene Therapy Candidate for the Treatment of Metachromatic Leukodystrophy

08/16/2022

– Candidate Showed Ability to Target Central Nervous System and Peripheral OrgansFollowing a Single I.V. Administration in MLD Model, a Key Differentiator from AvailableTreatments and Product Candidates –        – Data Demonstrated Biodistribution to Brain Regions and Multiple Cell Types –        – Optimizations Included Significant Improvements in Expression, Productivity and Packaging –        – Homology Seeks Partner…

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Integrated Medical Coding Module

07/03/2023

At TrialStat, we offer comprehensive medical coding services specifically tailored for clinical trials. Our advanced technology ensures fast and accurate coding of verbatim terms from both our Electronic Data Capture (EDC) system and external sources. With our innovative coding platform, we streamline the coding process, providing efficient and reliable solutions for your clinical research needs.…

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Incyte Announces Results of Phase 3 RUXCOVID Study of Ruxolitinib (Jakafi®) as a Treatment for Patients with COVID-19 Associated Cytokine Storm

12/24/2020

Excerpt from the Press Release: WILMINGTON, Del.–(BUSINESS WIRE)–Incyte (Nasdaq:INCY) today announced that the Phase 3 RUXCOVID study evaluating the safety and efficacy of ruxolitinib (Jakafi®), a JAK1/JAK2 inhibitor, plus standard-of-care (SoC) as a treatment for patients 12 years and older with COVID-19 associated cytokine storm did not meet its primary endpoint. Initial data show that…

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Trodelvy® Demonstrates Clinical Benefit for Black Patients Consistent with Full Metastatic Triple-Negative Breast Cancer Population in ASCENT Study

12/13/2021

– Disparities, Such as Access to Healthcare, Comorbidities and Differences in TNBC Disease Biology Typically Drive Worse Clinical Outcomes in Black Women Excerpt from the Press Release: FOSTER CITY, Calif.–(BUSINESS WIRE)–Dec. 10, 2021– Gilead Sciences, Inc. (Nasdaq: GILD) today announced new data from the Phase 3 ASCENT study evaluating Trodelvy® (sacituzumab govitecan-hziy) in relapsed or refractory metastatic triple-negative breast…

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Stoke Therapeutics Enrolls First Patient in a Natural History Study of People Living with Autosomal Dominant Optic Atrophy (ADOA)

08/31/2022

– ADOA is primarily caused by mutations in the OPA1 gene that result in progressive and irreversible vision loss in both eyes starting in the first decade of life – – The FALCON study is designed to evaluate disease progression and its effects on patients – – Data will support clinical development plans for STK-002,…

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U.S. Food and Drug Administration approves CSL’s HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B

12/01/2022

KING OF PRUSSIA, PA, USA This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion. Excerpt from the Press Release: This historic approval provides a new treatment option…

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BPGbio Announces Phase 2 BPM31510 Glioblastoma Trial-in-Progress Update at Society for Neuro-Oncology Annual Meeting

11/23/2023

Excerpt from the Press Release: BOSTON–(BUSINESS WIRE)–BPGbio, Inc., a leading biology-first AI-powered biopharma that focuses on oncology, neurology, and rare diseases, today announced that researchers will present data from an ongoing phase 2 trial study of BPGbio lead candidate, BPM31510, in patients with newly diagnosed glioblastoma multiforme (GBM), at the Society for Neuro-Oncology Annual Meeting…

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Caribou Biosciences Provides Regulatory Update on CB-010 Pivotal Plan with Phase 3 Trial Initiation Expected by YE 2024

12/14/2023

— Caribou met with the FDA and reached alignment on a pivotal trial in 2L LBCL with CB-010 versus a comparator arm of immunochemotherapy followed by HDCT and ASCT — — ANTLER Phase 1 trial continues dose expansion enrollment; initial dose expansion data and RP2D expected Q2 2024 — — Caribou plans to initiate Phase…

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Medical Coding Module

06/18/2023

Integrated Medical Coding Module Medical Coding Solutions for Clinical Trials At TrialStat, we offer comprehensive medical coding services specifically tailored for clinical trials. Our advanced technology ensures fast and accurate coding of verbatim terms from both our Electronic Data Capture (EDC) system and external sources. With our innovative coding platform, we streamline the coding process,…

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First AML Patient Transplanted with Vor Bio’s Trem-cel Demonstrated Durable Engraftment through Multiple Mylotarg™ Cycles at Initial Dose Level

03/02/2023

Excerpt from the Press Release: CAMBRIDGE, Mass., Feb. 16, 2023 (GLOBE NEWSWIRE) — Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, presented clinical data from VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (previously VOR33) in patients with acute myeloid leukemia (AML). In the first patient, trem-cel maintained hematopoiesis through three…

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