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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases. Genetic counselors are critical in this pathway, especially as more is learned about the human genome and relevant incidental findings.

Genetics 189
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4th Gene Therapy Analytical Development Summit 2022

pharmaphorum

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Download the full event guide to find out more.

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bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Regulatory Trends in Cell and Gene Therapies

Advarra

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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University of Edinburgh spinout Cellinta will develop gene therapies for cancer

The Pharma Data

. A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer. The funding will be used to develop a pipeline of ‘highly selective’ gene therapies targeting cancer stem cells in a variety of solid tumour types.