Worldwide Clinical Trials

NOVEMBER 27, 2023

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. Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel.

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Pharmaceutical Technology

JULY 14, 2022

AVR-RD-05 makes use of the own hematopoietic stem cells (HSCs) of a patient that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. Next year, Avrobio is expected to initiate a collaborator-sponsored Phase I/II clinical trial for Hunter syndrome in partnership with the University of Manchester, UK.

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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

SEPTEMBER 29, 2023

Immunotherapy has taken centre stage this week, with several announcements relating to regulatory approval, investment and research results for antibody and CAR-T therapies, perhaps most significantly, encouraging early-stage data for the first in vivo CAR-T therapy. The top stories: €27.3M

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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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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. In addition, I believe we will see more exosome-based therapies enter the clinic in 2024.

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Pharmaceutical Technology

APRIL 13, 2023

Significant collaborative R&D programmes that are likely to be affected include Takeda’s partnership with Codexis for AAV-based gene therapy for Fabry disease and Takeda’s $3.6bn dollar deal with Poseida for the development of nonviral in vivo gene therapy programmes for the hereditary bleeding disorder haemophilia A.

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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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Gene Therapy Drugs In-Vivo Clinical 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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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).

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BioTech 365

SEPTEMBER 29, 2020

First patient was administered today The trial aims at validating the safety of continuous administration of Ivermectin in oral form Simultaneously, several long-acting injectable formulations based on MedinCell’s BEPO® technology are being tested in vivo A 1-month active injectable prophylactic … Continue reading →

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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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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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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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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

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. Edinburgh-based PhaSER Biomedical (PhaSER) has received a Bill & Melinda Gates Foundation grant totalling $2.3m A key stage of compound progression. The post $2.3m

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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

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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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Pharmaceutical Technology

MAY 18, 2023

Other investors include Investissement Québec, Omega Funds, Cowen Healthcare Investments, CTI Life Sciences Fund III, Northleaf Capital Partners, BVF Partners and Vivo Capital. The combined company will be named enGene Holdings and its common shares are expected to be listed on the Nasdaq.

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Pharmaceutical Technology

APRIL 28, 2023

The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. g/dL and 45.4%

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Drug Discovery World

MAY 9, 2023

The recent passage of the FDA Modernization Act, which removed the requirement for animal testing when suitable alternatives are available, has highlighted the inadequacy of animal models most often used for in vivo research. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

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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

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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BioTech 365

SEPTEMBER 17, 2020

Positive results for Step-1 of Phase 3 clinical trial of Tedopi® in non-small cell lung cancer Positive preclinical and in human ex vivo results with CoVepiT vaccine against COVID-19; clinical entry expected by the end of 2020 or early 2021 … Continue reading →

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Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

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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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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. days quicker compared to placebo. days; 95% C.I, days; 95% C.I,

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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 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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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. Skin fibrosis is a leading feature of SSc associated with significant disability.

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pharmaphorum

JANUARY 13, 2021

IO Biotech, an oncology specialist formed and backed by Denmark’s Novo Holdings, has raised €127 million ($155 million) to further develop its cancer vaccine technology that has boosted efficacy of PD-1 immunotherapy in early trials. The post IO Biotech raises $155m to develop breakthrough cancer vaccine appeared first on.

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pharmaphorum

SEPTEMBER 28, 2022

It involves an ‘ex vivo’ use of gene-editing, with the technology is used to modify a patient’s own cells outside the body to make foetal haemoglobin (HbF), which can serve as a substitute to regular haemoglobin in both SCD and thalassaemia.

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The Pharma Data

JANUARY 17, 2021

Joe Dupere, CEO of Ixaka, commented: “Ixaka’s broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. A total of $15.4 A total of $15.4 References 1.

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The Pharma Data

MAY 3, 2021

DARS also greatly expanded its clinical trials operations, running three clinical trials related to streamlining biosimilars development and studying safety questions related to opioids and over-the-counter drugs,” Strauss wrote. Posted 03 May 2021 | By Michael Mezher . 2020 was a challenging year.

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Drug Discovery World

APRIL 5, 2023

A collaborative network The CBC operates as a collaborative network of 19 Institutes with longstanding drug discovery know-how from target identification through to proof-of-concept clinical trials. The post Only non-US company joins cancer drug discovery consortium appeared first on Drug Discovery World (DDW).

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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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