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.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In trials, Casgevy was shown to help prevent episodes of debilitating pain, known as vaso-occlusive crises, that often plague patients affected by sickle cell disease. In June 2023, the U.S.

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Drug Discovery World

FEBRUARY 20, 2024

One example is that the model can circumvent the difficulties in demonstrating in vivo drug efficacy which arise because of mouse specific metabolism issues. Gates Foundation grant to accelerate global drug discovery appeared first on Drug Discovery World (DDW). A key stage of compound progression. The post $2.3m

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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.

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Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

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XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.

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Drug Discovery World

APRIL 15, 2024

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 therapy will move to ex vivo gene modification and the delivery of gene-modified cell types. In addition, I believe we will see more exosome-based therapies enter the clinic in 2024.

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Drug Discovery World

MAY 9, 2023

Jantzen Sperry , PhD, Director of Scientific Operations at Certis Oncology looks at how the adoption of orthotopic PDX models can elevate the impact of cancer research and improve translation in oncology drug development. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

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Development Drugs In-Vivo Clinical Trials 52

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).

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Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

Clinical Trials 52

Clinical Trials Clinical Trials In-Vivo In-Vitro 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

Drug Discovery World

MARCH 15, 2024

million to accelerate development of its lead drug candidates in the area of mitophagy, MTX325 and MTX652, through clinical trials. from our investors, we can now make the next vital steps, progressing with essential clinical trials.” Mission Therapeutics has raised £25.2 ($32.1) Thanks to this additional £25.2m

Clinical Trials 52

Clinical Trials Clinical Trials In-Vivo Trials 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). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

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Gene Editing In-Vivo Gene Sequencing Gene 97

Drug Discovery World

APRIL 5, 2023

Italian research institute and contract research organisation IRBM has become the only company from outside the United States to join cancer drug discovery network the Chemical Biology Consortium (CBC). The post Only non-US company joins cancer drug discovery consortium appeared first on Drug Discovery World (DDW).

Drugs 52

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pharmaphorum

FEBRUARY 13, 2022

German drugmaker Merck KGaA has reached an agreement to pilot an artificial intelligence platform developed by Quris that aims to identify potential safety problems with new therapeutic candidates as early as possible in the drug discovery process.

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Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

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Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

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Roots Analysis

FEBRUARY 27, 2024

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6% during the forecast period 2023-2035. Get a complete list of the presentations, here.

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Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

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Development Drugs In-Vitro Gene Therapy 52

Drug Discovery World

JANUARY 18, 2024

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

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Drug Discovery World

OCTOBER 20, 2022

Aqemia has raised €30 million in a Series A funding round to further support its mission to massively scale drug discovery through a first-in-class technological platform combining quantum-inspired physics and machine learning. . We are continuously recruiting to address our next technology and drug discovery challenges”, she added. .

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pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

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Drug Discovery World

APRIL 11, 2023

12 The trial Tezepelumab was well tolerated in patients with severe asthma and resulted in clinically meaningful reductions in asthma exacerbations in NAVIAGTOR & SOURCE clinical trials and no clinically meaningful differences in safety results between the tezepelumab and placebo groups. to 0.53; P<0.001).3

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Clinical Trials Clinical Trials In-Vivo Medicine 98

Drug Discovery World

MAY 26, 2023

AnaMar has announced positive results from its Phase I study, and the completion of Phase II enabling pre-clinical studies, for AM1476’s initial orphan indication in systemic sclerosis (SSc) characterised by lung and skin fibrosis. The post Positive Phase I data for oral fibrosis treatment appeared first on Drug Discovery World (DDW).

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Camargo

APRIL 28, 2021

Conducting a clinical study in a pediatric patient population requires careful planning, as providing a benefit to a child without causing harm is a weighty responsibility. First Steps Toward Initiating Pediatric Clinical Studies. Data Collection from Preclinical and Adult Studies. Non-Linear Mixed Effect Modeling (NLMEM).

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The Pharma Data

MAY 3, 2021

In its 2020 annual report, the division of the US Food and Drug Administration (FDA) tasked with addressing emerging regulatory issues and developing new tools and approaches for regulatory reviews said it was able to expand its efforts despite the COVID-19 pandemic impacting most of the year. . Posted 03 May 2021 | By Michael Mezher .

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In-Vitro Clinical Trials Clinical Trials In-Vivo 52

pharmaphorum

JULY 22, 2021

We have completed phase 2 trial of a multi-antibody drug for influenza, CT-P27, and since 2015 we have been developing a therapeutic antibody to treat the Middle East Respiratory Syndrome (MERS), CT-P38. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19.

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Drug Discovery World

APRIL 8, 2024

Jakob Nikolas Kather, Professor of clinical artificial intelligence at Technical University Dresden in Germany, then discussed how AI applications can be used to derive prognostic and predictive information from routine pathology slides.

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Drug Discovery World

APRIL 4, 2023

The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. The drug was granted orphan drug designation by the FDA in May 2021. The drug is expected to become a standard of care for these patients, who have had limited treatment options, according to analysts GlobalData.

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FDA Approval In-Vivo Gene Editing Drugs 52

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.

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Drug Discovery World

DECEMBER 14, 2023

A number of companies presented breakthrough data for their investigational breast cancer drugs at the San Antonio Breast Cancer Symposium. We are thrilled to see this level of clinical activity in such a heavily pre-treated patient population,” said John Houston, Chairperson, Chief Executive Officer and President at Arvinas.

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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

Drug Discovery World

SEPTEMBER 14, 2023

The strategic R&D collaboration focuses on three pillars: Applying Moderna’s mRNA technology for in vivo expression of Immatics’ next-generation, half-life extended TCR bispecifics (TCER), targeting cancer-specific HLA-presented peptides.

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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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Gene Editing Gene Gene Therapy Drugs 59

Camargo

NOVEMBER 11, 2020

When the FDA requires a product’s labeling to include a boxed warning (also called a “black box warning” because the text is surrounded by black border), the potential market value of the drug often drops severely. Presumably, Antares’ long-established 90-person urology sales force can achieve pull-thru of this drug product.

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