XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

Gene Therapy 104

Gene Therapy Gene Trials Genetics 104

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. Continuing advancements within this field deliver new hope to doctors and patients, transforming disease outcomes for previously incurable indications. In the evolving biotechnology industry, enzymes are important process aids.

Drugs 295

Drugs Gene Therapy Manufacturing Production 295

Drug Discovery World

NOVEMBER 27, 2023

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

Production 59

Production Engineer Gene Editing Gene 59

pharmaphorum

SEPTEMBER 12, 2022

Across LabCorp , we innovate through science and technology, with access to approximately 2,500 [doctors] and PhDs and over 700 patients. Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Tailored oncology.

Genome 104

Genome Genomics Clinical Trials Clinical Trials 104

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 98

Genetic Disease Protein RNA Doctors 98

Drug Discovery World

APRIL 21, 2023

SLAS Student Poster Award The SLAS Student Poster Award recognises innovative research by students, graduate students, post- doctoral associates and junior faculty (less than four years in first academic appointment) who are chosen to present a poster during SLAS Europe 2023. It will be led by Björn C.

Doctors 52

Doctors Doctor Genetics In-Vitro 52

XTalks

APRIL 6, 2022

Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. If that happens, it is a big setback for patients and the doctors treating them,” said Dr. Rosenfeld in a news release by Cancer Research UK.

DNA 105

DNA In-Vitro Doctors Doctor 105

The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. Genetics / 12A. –( BUSINESS WIRE )– Stoke Therapeutics , Inc.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 9, 2020

Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate. He is currently the patient advocacy and engagement lead at Congenica, a digital health company that is investigating the genetic basis of rare diseases. For some people, a job is more than just a job.

Genome 86

Genome Genomics Genetics Genome Project 86

pharmaphorum

SEPTEMBER 9, 2020

Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate. He is currently the patient advocacy and engagement lead at Congenica, a digital health company that is investigating the genetic basis of rare diseases. For some people, a job is more than just a job.

Genome 75

Genome Genomics Genetics Genome Project 75

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Jonathan Fisher, PhD, Group Leader, University College London, on: ‘Local secretion of immune active proteins for direct and bystander tumour killing’.

Engineer 52

Engineer Antibody Protein In-Vivo 52

XTalks

AUGUST 10, 2021

This “SHERLOCK reaction” is designed to cut SARS-CoV-2 RNA at a specific region in the nucleoprotein gene that is conserved across multiple variants of the virus, and which codes for proteins involved in formation of the viral nucleocapsid. A porous membrane was engineered to capture RNA on its surface. miSHERLOCK Workflow.

RNA 105

RNA Containment DNA Engineer 105

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

Bioengineer

JULY 23, 2021

. “For the first time, we identified a crucial interaction between platelets and the tumor via their surface proteins,” Jain said. ” Jain’s team in College Station for this research includes postdoctoral researcher Dr. Biswajit Saha and doctoral students Jim Tronolone and Tanmay Mathur. ” ###.

Engineer 52

Engineer Gene Editing Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 21, 2021

Doctors need better tools to evaluate the status of COVID-19 patients as early as possible because many of the treatments—such as monoclonal antibodies—are in short supply, and we know that some patients will get better without intensive treatments,” said co-senior author Andrew E. The research was published in the journal JCI Insight.

Research 52

Research DNA Hormones Vaccination 52

The Pharma Data

MAY 15, 2021

Now imagine the entire length of the field as the 300,000 years of human genetic evolution. So you can see that your genes haven’t had time to adapt to the embarrassing over-abundance of food that you have today. However your genetic code IS hardwired to go without food for short periods between times of plenty.

Production 52

Production Hormones Protein Insulin 52

XTalks

SEPTEMBER 14, 2020

The company’s candidate vaccine, mRNA-1273, is a synthetic messenger RNA that encodes the stabilized SARS-CoV-2 spike protein. The University of Oxford/AstraZeneca partnership, in turn, is testing a viral-vectored coronavirus vaccine that again expresses the spike protein of SARS-CoV-2 virus.

Vaccination 92

Vaccination Vaccine Antibody Life Science 92