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 59

Gene Editing Gene In-Vivo Clinical Trials 59

Drug Discovery World

OCTOBER 11, 2022

A team of researchers at Northwestern University in the US has devised a new platform for gene editing that could inform the future application of CRISPR-based therapeutics. They created a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. High gene editing efficiency.

Gene Editing 52

Gene Editing Gene DNA Protein 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 59

Gene Editing Gene Gene Therapy Drugs 59

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. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

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.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

Gene Editing 105

Gene Editing Gene Life Science Regulation 105

BioSpace

OCTOBER 14, 2021

Read about TFF Pharmaceuticals and Augmenta Bioworks' dry powder COVID-19 antibody formula, the world's first gene editing clinical trial for PKU, Bayer's COVID-19 vaccine and other key developments in life sciences research.

Gene Editing 75

Gene Editing Life Science Clinical Trials Clinical Trials 75

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 97

Gene Editing In-Vivo Gene Sequencing Gene 97

Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

Gene Editing 52

Gene Editing Development Drugs Clinical Trials 52

Drug Discovery World

DECEMBER 8, 2023

“The future development of this combination therapy alongside our monotherapy clinical trials will help expand patient options and narrow the gap towards improving the long-term survival of patients with hepatocellular carcinoma.” Both companies will share the costs of manufacturing and preclinical assessments.

Antibody 52

Antibody Gene Editing Clinical Trials Clinical Trials 52

Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

Gene Therapy 111

Gene Therapy Gene Gene Editing Clinical Trials 111

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.,

Gene Editing 101

Gene Editing Genome Genomics Gene Therapy 101

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).

Gene Editing 100

Gene Editing Genetic Engineering Engineer Clinical Trials 100

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

MARCH 10, 2023

There have been several other announcements related to clinical trials in the last few days, including positive data from trials in severe influenza and biliary tract cancer, the launch of a study into an ADC treatment for solid tumours, and a new research partnership with the mission to improve clinical trial design.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 52

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.

Gene Editing 100

Gene Editing Clinical Trials Clinical Trials Genetics 100

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 Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

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.

Development 130

Development Gene Editing Gene Gene Therapy 130

Drug Discovery World

NOVEMBER 14, 2023

“I am very encouraged by the initial data from the heart-1 trial that demonstrated the potential for single-course gene editing as a new approach to treat patients with HeFH. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Gene Editing Gene Genome 52

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

DECEMBER 20, 2022

There will be greater emphasis on diversity in clinical trials. A focus on the representation of diverse patient populations in clinical trials will be a very good thing for the industry, but could be challenging. DDW’s Diana Spencer summarises the key trends likely to impact the market in 2023. .

Drugs 52

Drugs Gene Editing Clinical Trials Clinical Trials 52

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.

Genome 40

Genome Genomics Gene Gene Editing 40

Drug Discovery World

MAY 6, 2024

billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

Drugs 59

Drugs Life Science Gene Editing Gene 59

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

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

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.

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

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.

Research 118

Research Gene Editing Gene Genetics 118

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

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

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.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

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.

Gene Therapy 52

Gene Therapy Gene Gene Editing Clinical Trials 52

Drug Discovery World

MARCH 22, 2023

CRISPR gene editing The team used CRISPR-Cas9 gene editing to knock out Regnase-1 and Roquin-1 individually and together in healthy donor T cells with two different immune receptors that are currently being investigated in Phase I clinical trials: the mesothelin-targeting M5 CAR (mesoCAR) and the NY-ESO-1-targeting 8F TCR (NYESO TCR).

Gene Editing 52

Gene Editing Gene Engineer Regulation 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 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.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

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

Drug Discovery World

OCTOBER 24, 2023

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.

Gene Editing 52

Gene Editing Engineer Clinical Trials Clinical Trials 52

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.

Marketing 130

Marketing Gene Editing Production Sales 130

Drug Discovery World

SEPTEMBER 6, 2022

As we look to the future, many advanced therapy players are sticking to traditional cell and gene technologies like autologous cell therapies and gene therapy using adeno-associated viral vectors, but some are branching out to other models like allogeneic cell therapies, non-viral delivery methods and gene-editing technologies.

Gene Therapy 52

Gene Therapy Gene Gene Editing Manufacturing 52