pharmaphorum

MARCH 19, 2024

Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic

In-Vivo 52

In-Vivo Gene Editing Engineer Gene 52

Bio Pharma Dive

JANUARY 6, 2023

The Takeda-backed biotech revealed it’s raised $85 million in new funding alongside announcing the acquisition of gene editing startup Twelve Bio.

In-Vivo 304

In-Vivo Gene Editing Gene 304

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Bio Pharma Dive

OCTOBER 25, 2023

The study results, while only from three participants, offer a glimpse at how one of the few in vivo CRISPR therapies in U.S. human testing is working.

Gene Editing 148

Gene Editing In-Vivo Gene 148

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We This is a defining time for Eligo as this funding puts us in a strong position to make a significant leap in treating diseases by editing the genetic makeup of the human microbiome.”

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

BioSpace

APRIL 24, 2024

Regeneron will use Mammoth Biosciences’ tiny Cas enzymes to deliver in vivo CRISPR-based gene editing therapies to tissues and cell types beyond the liver, the companies announced Thursday.

Gene Editing 99

Gene Editing In-Vivo Gene 99

BioPharma Reporter

JANUARY 10, 2022

have announced a strategic collaboration and option agreement for the use of Mammothâs CRISPR systems to develop in vivo gene-editing therapies. Bayer AG and Mammoth Biosciences, Inc.,

Gene Editing 84

Gene Editing Gene In-Vivo Development 84

BioSpace

JULY 6, 2021

This appears to be a second example of CRISPR gene editing inside the body, as opposed to in cell cultures.

Gene Editing 104

Gene Editing In-Vivo Gene Medicine 104

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing 52

Gene Editing Genetic Disease Gene In-Vivo 52

BioTech 365

AUGUST 25, 2021

Precision BioSciences to Host In Vivo Gene Editing R&D Event on September 9, 2021 Precision BioSciences to Host In Vivo Gene Editing R&D Event on September 9, 2021 DURHAM, N.C.–(BUSINESS –(BUSINESS WIRE)–Precision BioSciences, Inc.

Gene Editing 40

Gene Editing In-Vivo Gene 40

BioSpace

OCTOBER 18, 2023

The trial, which is the first late-stage study of an in vivo CRISPR treatment in the U.S., will start by the end of 2023. Intellia's NTLA-2001 is a treatment candidate for transthyretin amyloidosis cardiomyopathy.

Gene Editing 111

Gene Editing In-Vivo Gene Trials 111

BioTech 365

OCTOBER 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Intellia, LogicBio fuel hope for in vivo gene editing with new animal data in rare diseases: report.Intellia, LogicBio fuel hope for in vivo gene editing with new … Continue reading →

Gene Editing 40

Gene Editing In-Vivo Gene Research 40

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

JANUARY 10, 2024

Precision BioSciences licenses non-cancer uses of its allogeneic CAR-T therapy azer-cel to TG Therapeutics as it pivots to in vivo applications of its gene-editing technology

Gene Editing 111

Gene Editing In-Vivo Licensing Licensing 111

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

Gene Editing 59

Gene Editing Gene Gene Therapy Drugs 59

Drug Discovery World

NOVEMBER 14, 2023

It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

In-Vivo 52

In-Vivo Gene Editing Gene Genome 52

BioSpace

AUGUST 3, 2021

With this milestone, the field is now wide open for the next in vivo CRISPR success, and Editas is hoping that it will be in the ocular disease space.

Gene Editing 134

Gene Editing In-Vivo Gene 134

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 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

BioTech 365

MAY 1, 2021

Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting Editas Medicine to Present Preclinical Data Demonstrating Advancements … Continue reading →

Gene Editing 40

Gene Editing In-Vivo Genetics Gene 40

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

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

BioTech 365

JANUARY 12, 2021

Verve Therapeutics Announces Data Demonstrating Durable LDL Cholesterol Lowering After a One-Time Gene Editing Treatment in Non-Human Primates Verve Therapeutics Announces Data Demonstrating Durable LDL Cholesterol Lowering After a One-Time Gene Editing Treatment in Non-Human Primates Results Validate In Vivo … Continue reading →

Gene Editing 52

Gene Editing Gene In-Vivo 52

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

Genome 40

Genome Genomics Gene Gene Editing 40

BioSpace

OCTOBER 25, 2021

Mammoth Biosciences just announced a collaboration with Vertex to develop in vivo gene-editing therapeutics for patients with either of two serious, but not-yet-disclosed diseases.

Gene Editing 90

Gene Editing In-Vivo Gene Development 90

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. g/dL and 45.4%

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body — found that the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

DNA 142

DNA Gene Editing In-Vivo Genetics 142

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

NOVEMBER 18, 2022

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial. LCA10 is caused by mutations in the CEP290 gene and accounts for approximately 20?30% 30% of all LCA cases.

Sales 92

Sales Trials In-Vivo Gene Editing 92

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

APRIL 15, 2024

Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types. DDW’s Megan Thomas speaks to experts from the drug discovery industry about their predictions on the future of stem cells in drug discovery.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Pharmaceutical Technology

FEBRUARY 15, 2023

The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells. CRISPR nucleases serve as an important genome editing tool. However, not all innovations are equal and nor do they follow a constant upward trend.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Vertex is also offering up to $1.3

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

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In-Vivo FDA Approval Gene Gene Editing 80

Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

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In-Vivo Clinical Trials Clinical Trials Drugs 52