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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Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

World of DTC Marketing

JANUARY 31, 2021

I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases.

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In-Vivo Generic Drugs Drugs Gene Therapy 187

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.

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Gene Editing Gene Gene Therapy In-Vivo 130

pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt. Vertex is also offering up to $1.3

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

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Gene Splicing Gene DNA Recombinant DNA Technology 130

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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

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

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.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

OCTOBER 6, 2022

By reducing the disease to its key components, a computer-readable network of disease markers was characterised using genetic, biomarker, and clinical data; the model was also populated with ‘disease vs control’ data. Disease modelling Gulf War Illness. Tinker, tailor, soldier, therapy.

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In-Vivo Research Genetics Drugs 98

pharmaphorum

OCTOBER 8, 2020

Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. 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.

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Gene Editing Gene In-Vivo Clinical Trials 69

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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Gene Therapy Gene In-Vivo Genetics 40

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

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.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

AUGUST 24, 2021

It will focus on ‘ex vivo’ therapies, which involves harvesting cells from patients, modifying them and then infusing then back into the body. “This new collaboration further expands our toolkit in cell and genetic therapies,” said Bastiano Sanna, Vertex’ chief of cell and genetic therapies. .

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy Gene FDA Approval In-Vivo 98

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

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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In-Vitro Clinical Trials Clinical Trials In-Vivo 52

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. They are due to enrol 45 apiece, and are scheduled to readout next year.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

XTalks

AUGUST 17, 2020

With several biologics in the pipeline, including one in early stage clinical trials for head and neck cancer, the company is positioned to engineer highly specific biologics against immune targets in the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.

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Protein Engineer Immune Response In-Vivo 98

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccine 52

Vaccine Vaccination In-Vivo Genetics 52

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. Related: Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug The advisors appeared convinced that exa-cel can offer significant benefits to people with sickle cell. I think in this case, there is a huge unmet need.”

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Gene Therapy Gene Gene Editing In-Vivo 59

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 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.

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Licensing Licensing Antibody Gene Therapy 52

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869). About Lysogene.

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

Roots Analysis

AUGUST 23, 2024

Unlike traditional forms of vaccines modern day vaccines use the virus’ genetic code itself to give our cells instruction on how to produce this antigen molecule, meaning we only ever need a tiny amount of an antigen in the cure for it work. Ribosomes are cellular machines that read mRNA sequences and produce proteins.

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Protein Vaccination Vaccine Immune Response 40

The Pharma Data

APRIL 8, 2022

Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19. Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19.

Immune Response 52

Immune Response In-Vivo Genetic Analysis Research 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The company was running Phase III trials of the therapy for AS; however, the therapy flunked in the trial failing to meet its primary endpoint. As a consequence, Ovid halted everything related to OV101 and Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Nielsen, the primary inventor of peptide nucleic acid (PNA) technology, brings extensive experience in genetic medicine to the company.

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In-Vivo Business Development Manufacturing Development 52

Roots Analysis

AUGUST 31, 2023

An additional level of genetic control is provided by gene switches that are located upstream of the promoter region. These genetic switches assist transcription factors in binding to the promoter region. This is true for any genetic products or byproducts of the metabolic pathways that are toxic to the host.

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Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

JULY 8, 2021

8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) 8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) Amgen (NASDAQ:AMGN) announced that the U.S.

Allergies 52

Allergies Trials In-Vivo Medicine 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

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Engineer Containment In-Vivo Production 52

Roots Analysis

SEPTEMBER 5, 2023

Further, owing to lack of understanding of how drug molecules were likely to behave in vivo , many lead molecules that progressed into the clinical stages of development had to be discontinued when they failed to demonstrate adequate therapeutic benefit.

Drugs 52

Drugs In-Vivo Development Research 52

XTalks

OCTOBER 1, 2020

The drug is currently in clinical trials for alcoholic hepatitis (AH), nonalcoholic steatohepatitis (NASH), as well as in COVID-19 patients with acute liver or kidney injury. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments. A life-changing moment came for Dr.

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Scientist In-Vitro Research Life Science 52