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

Pharmaceutical Technology

APRIL 26, 2024

Regeneron Pharmaceuticals has collaborated with Mammoth Biosciences to develop in vivo clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing therapies.

Gene Editing 130

Gene Editing Gene In-Vivo Development 130

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Bio Pharma Dive

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

Gene Editing 190

Gene Editing In-Vivo Gene Medicine 190

Bio Pharma Dive

JANUARY 22, 2025

The biotech said it has dosed the first participant in a Phase 3 study of its in vivo gene editing treatment. Elsewhere, the FDA imposed a hold on Atara and lifted another on Amylyx.

Gene Editing 176

Gene Editing In-Vivo Gene Drugs 176

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 157

Gene Editing In-Vivo Gene 157

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Topic sponsors are not involved in the creation of editorial content.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

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 96

Gene Editing In-Vivo Gene Medicine 96

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 92

Gene Editing Gene In-Vivo Development 92

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 132

Gene Editing In-Vivo Gene 132

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

BioSpace

APRIL 29, 2024

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

Gene Editing 88

Gene Editing Gene Medicine In-Vivo 88

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

Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The applications include requests for priority review. It is being assessed for the treatment of SCD or TDT patients.

Gene Therapy 246

Gene Therapy Gene Editing In-Vivo Gene 246

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

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 96

Gene Editing In-Vivo Gene Trials 96

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

pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

Gene Editing 111

Gene Editing In-Vivo Drugs Clinical Trials 111

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 94

Gene Editing In-Vivo Gene 94

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

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

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

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

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

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

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

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

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

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

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 76

Gene Editing In-Vivo Gene Development 76

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

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 Genomics Genome Gene Editing 162

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

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 40

Gene Editing Gene In-Vivo 40

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 In-Vivo Trials Gene Editing 92

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 Gene FDA Approval In-Vivo 98

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105