article thumbnail

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

RNA 245
article thumbnail

Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

XTalks

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. Related: Red Meat Allergy Test Gets FDA Clearance. “We COVID-19 Clinical Trials.

Insiders

Sign Up for our Newsletter

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

article thumbnail

Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and gene expression patterns. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

article thumbnail

Tezepelumab NAVIGATOR Phase 3 Trial Met Primary Endpoint Of A Statistically Significant And Clinically Meaningful Reduction In Exacerbations In A Broad Population Of Patients With Severe Asthma

The Pharma Data

AstraZeneca continues to lead development and Amgen continues to lead manufacturing. About Amgen Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics.

Trials 52
article thumbnail

Gene editing: beyond the hype

pharmaphorum

It has also built its own manufacturing site and launched partnerships with six big pharma companies. This growth reflects the continued and increasing interest in gene therapy – and with stock prices rising for editing companies across the board, Macrae says there has never been a more interesting time to be in genomic medicine.

article thumbnail

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

RNA 40
article thumbnail

Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation

The Pharma Data

Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. Manufacturing cells is not like manufacturing small molecules,” Brian Culley, CEO of Lineage Cell Therapeutics , told BioSpace.