AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.

Genetic Disease 189

Genetic Disease Genetics Gene Editing Medicine 189

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.

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Gene Editing Gene Medicine In-Vivo 88

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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Gene Editing In-Vivo Drugs Clinical Trials 111

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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Gene Editing Gene Gene Therapy Drugs 59

Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial.

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Gene Therapy Gene Editing In-Vivo Gene 246

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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Gene Editing Genomics Genome Gene Therapy 101

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. The Therapeutic Strategy Leads for Rare Disease at Worldwide Clinical Trials have been working alongside and championing these organizations for many years.

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Gene Gene Editing Gene Therapy Clinical Trials 130

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

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Gene Editing In-Vivo Gene Sequencing Gene 97

Pharmaceutical Technology

APRIL 28, 2023

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin.

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Gene Therapy Drugs Gene Gene Editing 264

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome.

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Gene Editing DNA Gene Genome 98

Pharmaceutical Technology

OCTOBER 13, 2022

Vita Therapeutics intends to use the funding to advance its lead pre-clinical programme VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to clinical trials. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The company has raised nearly $66m in total since its inception.

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Development Gene Editing Gene Gene Therapy 130

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. million) Nobel Prize award. “In

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Gene Editing Bacteria DNA Gene 105

Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The company develops gene-editing organ transplantation technology. QN-023a overview QN-023a is under development for the treatment of relapsed or refractory acute myeloid leukemia (AML).

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The company develops gene-editing organ transplantation technology. Hangzhou Qihan Biotechnology overview Hangzhou Qihan Biotechnology is a healthcare company.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

WCG Clinical

OCTOBER 21, 2024

Clinical trials are the cornerstone of advancing medical research, but behind the science, the experiences of participants and their families offer invaluable insight into how research impacts real lives. Advances in research, such as gene editing and cellular therapies, have opened new treatment possibilities for both conditions.

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Clinical Trials Clinical Trials Trials Clinical Research 52

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.

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Gene Editing In-Vivo DNA In-Vitro 105

pharmaphorum

SEPTEMBER 29, 2020

As summarised by The National Institutes of Health, this includes reducing the number of redundant clinical trials, enhancing the statistical strength of studies, reducing overall costs and risks, and improving study participant recruitment, all while triggering creativity and innovation between collaborators.

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Research Gene Editing Development Gene 132

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems.

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Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

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.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

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

Pharmaceutical Technology

JANUARY 19, 2023

While allogeneic hematopoietic stem cell transplants (HSCT) provide long-term benefit to a subset of AML patients, other cell therapy modalities such as natural killer (NK) cells or chimeric antigen receptor (CAR)-T cells have failed to show conclusive benefit in late-stage clinical trials.

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Marketing Gene Editing Production Sales 130

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. Clinical trials have demonstrated the potential of CRISPR/Cas9 in this field.

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Research Gene Editing Gene Genetics 118

pharmaphorum

NOVEMBER 23, 2021

It is also close to Cambridge University, seen as one of the world’s leading life sciences research centres, as well as the city’s Addenbrookes Hospital, an important centre for clinical trials in the National Health Service.

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Gene Editing Life Science Pharma Companies Clinical Trials 105

pharmaphorum

MARCH 19, 2021

Around $7 to $9 million from the proceeds of the IPO will be used to advance Gain’s preclinical-stage candidates for Morquio and GM1 into phase 1/2 clinical trials, according to the prospectus for the offering. The post Protein misfolding specialist Gain nets $40m from IPO appeared first on.

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Protein Gene Editing Drugs Clinical Trials 103

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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In-Vivo Gene Editing Trials Gene 52

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.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs.

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Gene Therapy Gene Gene Editing Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced.

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Gene Therapy Gene Gene Editing Clinical Trials 52

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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Gene Editing Manufacturing Gene In-Vivo 52

Delveinsight

SEPTEMBER 14, 2021

The proceeds from the Series B financing will be utilized to progress ADARx’s proprietary RNA platform technologies for base editing, inhibition, degradation, and delivery. Vor said in a statement that it is actively enrolling in its Phase I/II clinical trial for AML patients who currently have restricted treatment options.

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67

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.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

The Pharma Data

AUGUST 4, 2020

Under the terms of the deal, Oxford Biomedica will receive an undisclosed upfront payment, with additional payment related to development and manufacturing of lentiviral vectors for use in clinical trials.

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Gene Editing Licensing Licensing Clinical Supply 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Research Drugs Gene Therapy Clinical Trials 59

Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated.

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Genome Genomics Gene In-Vivo 40

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. The potential for CRISPR gene editing to offer a therapeutic option to patients is clear, based on its mechanism, but there are already gene editing therapeutics on the market.

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Gene Editing DNA Gene Genetics 52