pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 97

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

Drug Discovery World

DECEMBER 12, 2022

The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for target validation, and its applications in therapeutic genome editing. Register here.

Genome 52

Genome Genomics Engineer In-Vivo 52

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. Photo courtesy of Science Advances. ” Source link.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

DNA 98

DNA Genome Genomics RNA 98

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– EdiGene, Inc. as Head of Business Development. “Our

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site.

DNA 52

DNA In-Vivo Genetics Medicine 52

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. CAMBRIDGE, Mass., 11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52