article thumbnail

To Share or Not to Share Failed Genetic Screening Results with Patients

Worldwide Clinical Trials

The best course of action will depend on many variables, such as the disease or population being studied and the genotype/phenotype relationship, risk, and impact on care. Obtaining appropriate assistance to facilitate planning up front for the complexities involved will save sponsors time and effort in the long run.

Genetics 130
article thumbnail

Risk adjusted net present value: What is the current valuation of Pharma Mar’s Ecubectedin?

Pharmaceutical Technology

The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress. Pharma Mar offers in vitro diagnostic products used for detection of virus and bacteria in respiratory infections and human papilloma virus genotypes, among others.

Hormones 100
Insiders

Sign Up for our Newsletter

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

article thumbnail

The biotech improving immunotherapies with AI

Drug Discovery World

Where cancers were previously understood by their tissue of origin and cell morphology, we can now define them based on their genotypes and molecular drivers. We are building a map of tumour immunotypes as the basis for identifying therapeutically relevant biology that translates from discovery to clinical development.

Genome 52
article thumbnail

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. 0 genotypes. The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0

article thumbnail

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

For rare disease studies where there is little or no clinical trial experience, outcome assessments would have to be validated within a clinical development program. Early and ongoing engagement with regulators greatly helps clinical development planning.

article thumbnail

Global Roundup: Overland Pharma Launches in China to Provide Access to Breakthrough Therapies

The Pharma Data

The aim of the research will be to create a TGS IVD platform by optimizing a multi-layer approach encompassing the initial sample and library preparation, state-of-the-art sequencing technologies and improved genotyping procedures. Amolyt has also acquired an option to license the identified candidates for future clinical development.

In-Vivo 52
article thumbnail

How biomarkers can help discover new treatments for women’s health

Drug Discovery World

RA: You’ve recently announced a collaboration with the University of Oxford to access genotype data on women with endometriosis. We previously conducted a hypothesis-free study using genotype data from the UK Biobank, which resulted in the first mechanism-based stratification of endometriosis patients.

Genome 72