The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Delveinsight

JANUARY 14, 2021

Both companies retain full rights to their product candidates and discuss the potential path forward for any future combination studies based on the outcome of the Phase 2 trial. Eligible members will get an integrated product experience, comprising the Livongo for Diabetes program, Dexcom CGM technology, and CGM-powered insights at no cost.

Gene Editing 40

Gene Editing Gene Genetics DNA 40

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. to 10-fold relative to baseline within 28 days after the cell product infusion. percent in the placebo group.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccination Vaccine 98

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. SATURDAY, Dec. “This is similar to bone marrow transplantation. Food and Drug Administration.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

Gene Editing 98

Gene Editing Gene DNA Engineer 98

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The biotech is focused on mRNA but has developed a self-amplifying mRNA (sa-RNA) platform in an attempt to develop the potential of the technology further.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

Worldwide Clinical Trials

MAY 30, 2024

was withdrawn, rendering it unapproved for patient use, based on negative review comments by the Committee for Medicinal Products for Human Use. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient. The MAA file in the E.U.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

Pharmaceutical Technology

FEBRUARY 20, 2023

Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors. The opportunity here for our expanded CRO to advance personalised medical solutions is huge.

Research 130

Research Clinical Trials Clinical Trials Trials 130

XTalks

AUGUST 2, 2023

Most of the company’s revenue comes from the manufacture and sale of biopharmaceutical products. They are committed to capitalizing on growth opportunities primarily through the advancement of their own product pipeline and constantly improving their existing products, as well as through business development activities.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

XTalks

SEPTEMBER 21, 2020

A female’s second X chromosome is typically silenced (so that females don’t get an overdose of X chromosome genes) through X-linked inactivation, a process where the chromosome is coated with an outer layer of non-coding RNA. One of these doubly active genes is KDM6A.

Gene 89

Gene Protein Gene Expression Scientist 89

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccination Vaccine Gene 111

XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111