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 The funding is earmarked for accelerating the development of Eligo’s flagship programme, EB005, which targets moderate to severe acne vulgaris.

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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.

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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.

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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. FH is one of the most common genetic conditions, affecting around one in 300 people globally.

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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.

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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.

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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

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Gene Editing Gene In-Vivo Genetics 52

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). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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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.

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Gene Editing In-Vivo Gene Development 88

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.

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Gene Editing Gene In-Vivo Clinical Trials 54

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%

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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.

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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?

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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.

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Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

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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., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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Gene Therapy Gene Gene Editing In-Vivo 40

Drug Discovery World

AUGUST 4, 2023

Charles River Laboratories has revealed plans to open Charles River Accelerator and Development Lab (CRADL) locations in Seattle and Philadelphia, expanding the company’s contract vivarium space for the biopharmaceutical industry. Both Seattle and Philadelphia are emerging as cities with significant biopharmaceutical communities.

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Life Science Gene Therapy Gene Editing In-Vivo 52

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

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Gene Editing Genetics Drugs Gene 52

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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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.

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Gene Editing Clinical Trials Clinical Trials In-Vivo 59

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. The post Could CRISPR cure HIV?

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Gene Editing In-Vivo DNA In-Vitro 105

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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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., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

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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). Lilly will provide funding for the Phase I clinical trials.

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Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. What are organoids? .

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Gene Editing Gene Drugs In-Vivo 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.

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Gene Therapy Gene Gene Editing In-Vivo 59

Drug Discovery World

APRIL 4, 2023

Read more: New oral option for anaemia of CKD approved in US EA-2353, Endogena Therapeutics The investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) was designated as a Fast Track development programme in February. Here’s a summary. The drug was granted orphan drug designation by the FDA in May 2021.

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FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

MARCH 9, 2023

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW). Intellia’s multi-national Phase I/II study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE.

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Drug Discovery World

JULY 3, 2023

This was centred around pre-clinical models (in vitro and in vivo) to support discovery and development, re-entering the fray with costimulatory agonists, and clinical development strategies for combinations with immune cell engagers​.

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Antibody In-Vitro Engineer In-Vivo 52

Pharma Marketing Network

DECEMBER 21, 2020

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. Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1.

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Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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Gene Therapy FDA Approval Gene In-Vivo 98

Delveinsight

FEBRUARY 25, 2021

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. Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence.

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Drug Discovery World

JUNE 7, 2023

DDW’s Diana Spencer chats to Macomics’ co-founder and VP Immunology Dr Luca Cassetta about the company’s approach to tumour suppression and the challenges of launching a new business in early 2020. DS: Congratulations on the publication of your discoveries in Nature Reviews Cancer. Your studies focus on tumour associated macrophages (TAMs).

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Drug Discovery World

NOVEMBER 16, 2022

Therefore, while researchers can understand just how a drug molecule impacts a cell, they can’t be sure that it will perform the same when used in-vivo. . One of these benefits is how cells grown in this type of culture are able to interact with their surroundings, similar to how they would perform in-vivo. 2D vs 3D .

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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.

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Drug Discovery World

APRIL 3, 2023

DS: What motivated you and Dr David Bunka, CTO, to create Aptamer and did you imagine you would one day be operating on such a global scale?    I quickly recognised the commercial value of aptamers as being able to address the gap in the market where antibodies fail to perform, and together we established the business to begin aptamer development.

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