Remove Presentation Remove Treatment Remove Trials
article thumbnail

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Clinical trials are expensive, slow and often limited by outdated design constraints.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.

article thumbnail

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.

Disease 80
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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 105
article thumbnail

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.

article thumbnail

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.

article thumbnail

BMS Presents Transcend FL Data: Breyanzi Shows Durable Responses in Marginal Zone Lymphoma

The Pharma Data

Currently, the medial survival for patients with MZL after multiple lines of treatment falls within the range of 3–5 years, reflecting a significant and persistent need for more effective therapeutic options. We are proud to present for the first time the primary analysis data from the MZL cohort of TRANSCEND FL. Lia Palomba, M.D. ,

article thumbnail

Merck’s WINREVAIR Succeeds in Phase 3 Trial for Pulmonary Arterial Hypertension

The Pharma Data

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Primary Endpoint and Clinical Results The HYPERION trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in the risk of time to clinical worsening (TTCW).

Trials 40