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

Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields.

Gene Therapy 52

Gene Therapy Gene Drugs In-Vivo 52

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment.

In-Vivo 246

In-Vivo Gene Therapy Gene RNA 246

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

Gene Editing 144

Gene Editing Gene Gene Therapy In-Vivo 144

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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. g/dL and 45.4%

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

BioSpace

NOVEMBER 18, 2021

Avrobio’s ex vivo lentiviral gene therapy platform led to durable responses measured in years, and significant reductions in disease-related biomarkers that far exceed today’s standard of care.

In-Vivo 98

In-Vivo Gene Therapy Gene Trials 98

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 investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 130

Gene Therapy Gene In-Vivo Life Science 130

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

Roots Analysis

FEBRUARY 27, 2024

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. The global genome editing market is anticipated to grow at a CAGR of 12.6%

Genome 40

Genome Genomics Gene Gene Editing 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 86

Gene Editing In-Vivo Gene Sequencing Gene 86

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).

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 52

Pharmaceutical Technology

MAY 24, 2023

is expected to enter Phase II/III registration trial as the first-line treatment for advanced Merkel cell carcinoma (MCC) early next year. to investigational new drug-enabling trials, expected in 2024, to treat aggressive diffuse large B-cell lymphomas (DLBCLs). Morphogenesis’ lead personalised cancer vaccine, IFx-Hu2.0,

Gene Therapy 130

Gene Therapy In-Vivo In-Vitro Immune Response 130

Drug Discovery World

MAY 29, 2023

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone.

Gene Therapy 52

Gene Therapy In-Vivo Gene Sales 52

Drug Discovery World

MARCH 13, 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). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

Gene Therapy 52

Gene Therapy Drugs In-Vivo Clinical Trials 52

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

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 58

Gene Editing Gene Gene Therapy Drugs 58

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 58

Gene Editing Gene In-Vivo Clinical Trials 58

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

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD). Patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.

FDA Approval 52

FDA Approval Trials Radiology In-Vivo 52

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

Drug Discovery World

MARCH 13, 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). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

In-Vivo 52

In-Vivo Gene Therapy Clinical Trials Clinical Trials 52

Drug Discovery World

MARCH 13, 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). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

In-Vivo 52

In-Vivo Gene Therapy Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. In a recent webinar, Dr. Vassallo discussed the operational considerations for complex cell therapy clinical trials.

Clinical Trials 52

Clinical Trials Clinical Trials Trials 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

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).

In-Vivo 80

In-Vivo FDA Approval Gene Gene Editing 80

Drug Discovery World

JUNE 30, 2023

This pairing includes reprogrammed cells for both wild and mutation-specific Huntington’s Disease (H), Amyotrophic Lateral Sclerosis (ALS), and Alzheimer’s Disease (AD) and is expanding to include custom disease-relevant mutations. Disease relevant in vitro assays improve translation in vivo, leading to clinical success.

Development 52

Development Drugs In-Vitro Gene Therapy 52

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

Gene Therapy 40

Gene Therapy Gene In-Vivo Clinical Trials 40

Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes.

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

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.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Delveinsight

JANUARY 12, 2021

million after Birinapant successfully becomes a part of the Phase I trials. The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. The duo will jointly develop the therapy, conducting clinical trials globally.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

JUNE 14, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. Gene-modified MSC were presented as an example of MSC 2.0.

In-Vivo 52

In-Vivo Gene Manufacturing Development 52