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The AI model that is changing clinical trial design

Drug Target Review

Clinical trials are expensive, slow and often limited by outdated design constraints. These digital twins are created for each trial participant using their baseline data – regardless of whether they are assigned to the placebo or treatment arm – and simulate how that individual would have responded under control conditions.

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. While commonly used in oncology, the basket trial approach is novel for rare genetic diseases.

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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New cell therapy model accelerates cancer treatment development

Drug Target Review

In just two years, CTMC has advanced eight therapies into clinical trials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. This circular process presents unique challenges, but the model Bock has developed at CTMC is tailored to overcome these hurdles efficiently.

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Managing CGT trials: the role of IRT from discovery to clinical development

Drug Target Review

The journey of cell and gene therapies from preclinical discovery to clinical trials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. These digital platforms are designed to manage and automate critical trial processes, especially related to drug management.

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The rising impact of biomarkers in early clinical development

Drug Target Review

A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms.

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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

FDA