BioTech 365

MAY 1, 2021

Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting Editas Medicine to Present Preclinical Data Demonstrating Advancements … Continue reading →

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Gene Editing In-Vivo Genetics Gene 40

pharmaphorum

JULY 5, 2022

Cell therapy specialist Mogrify has struck a deal with Japanese drugmaker Astellas to look at ways to deploy regenerative medicine to treat hearing loss caused by factors such as chronic exposure to loud noises. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide.

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In-Vivo Bioinformatics Genetics Licensing 110

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

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Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

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In-Vivo Gene Editing Trials Gene 52

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . These programmes will include therapies and vaccines in infectious disease and oncology areas.

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RNA Vaccine Vaccination In-Vivo 147

Scienmag

JULY 18, 2021

Together these Oncotarget results describe the first ever CAC case with a BRCAness genetic background Credit: Correspondence to – Juha K.

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In-Vivo DNA Genetics Medicine 89

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.

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Genetics Medicine Gene Therapy Marketing 52

Pharmaceutical Technology

MAY 24, 2023

The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine. CGT drugs fall into two major categories: autologous and allogeneic. What are the general differences in the supply chain of CGT vs. traditional clinical trials?

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

BioTech 365

OCTOBER 6, 2020

Built by CRISPR leaders including Jennifer Doudna to enable best-in-class in vivo therapeutics that permanently treat the underlying cause of disease Initial $20 million Series A led by Andreessen Horowitz Collaboration with Biogen Inc. focused on development of novel genetic … Continue reading →

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Genetics In-Vivo Engineer Medicine 40

World of DTC Marketing

JANUARY 31, 2021

Deloitte goes on to say “while biologics generally target smaller populations, “next-gen” therapies often target individual patients—sometimes as single treatments—resulting in truly personalized medicine. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. percent from 2018.

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In-Vivo Generic Drugs Drugs Gene Therapy 187

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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Gene Editing Genetics Drugs Gene 52

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). The company will receive a combined upfront payment and equity investment of $60m.

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Gene Editing Gene Gene Therapy In-Vivo 130

Scienmag

NOVEMBER 12, 2020

Gong Chen at Jinan University, Guangzhou, China recently reported the first non-human primate study demonstrating successful in vivo neural regeneration from brain internal glial cells for stroke repair. Credit: Jinan University Stroke is a leading cause of death and severe long-term disability with limited treatment available.

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Gene Therapy In-Vivo Gene Research 98

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

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. CAMBRIDGE, Mass.,

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Scienmag

OCTOBER 20, 2020

Credit: CNIO It has recently been described that infection by some enteroviruses – a genus of viruses that commonly cause diseases of varying severity – could potentially trigger diabetes, although its direct effect ‘in vivo’ as well as its mechanism of action at the molecular level were unknown.

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In-Vivo Research Gene Genetics 68

The Pharma Data

DECEMBER 13, 2020

Looking ahead, our top priority remains focused on advancing our allogeneic CAR T cell therapy and in vivo gene correction pipelines to speed development of medicines for unmet needs in cancer and rare diseases.”. About Precision BioSciences, Inc. Precision BioSciences, Inc.

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In-Vivo Genome Genomics Gene 52

Scienmag

AUGUST 5, 2020

5, 2020) — Genetic modifier HDAC6 was found to control tumor growth and halt metastasis in triple-negative breast cancer in vivo, according to a new study published […].

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In-Vivo Genetics Drugs Research 41

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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DNA In-Vivo Genetics Medicine 52

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

Scienmag

NOVEMBER 16, 2020

Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

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In-Vivo RNA DNA Genome 53

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2

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Gene Editing Gene In-Vivo Genetics 52

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. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies. Benjamin Oakes.

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Gene Editing Gene In-Vivo Clinical Trials 69

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Rare diseases can often be progressive, chronic and fatal. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

APRIL 12, 2021

C4XD said it has developed a molecule small enough to be formulated into a pill, which has also been able to block IL-17 in vivo. This follows a tie-up between C4XD, Sano Genetics and BenevolentAI to accelerate research into inflammatory bowel disease, which was signed in February.

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Drugs In-Vivo Licensing Licensing 52

pharmaphorum

JANUARY 17, 2023

Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential. The pandemic proved that mRNA technology could provide effective protection against infectious disease, at least in the case of COVID-19.

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Vaccination Vaccine In-Vivo In-Vitro 97

The Pharma Data

NOVEMBER 22, 2020

Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. “Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to edit,” George Church, a professor of genetics at Harvard Medical School, told the news source.

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Gene Editing DNA Genome Genomics 52

Roots Analysis

JANUARY 27, 2025

Cell therapies are based on the premise that the patients own cells (autologous), or those from a healthy donor (allogeneic), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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Manufacturing Contamination Production Gene Therapy 40

Scienmag

FEBRUARY 22, 2021

Using innovative in vivo imaging approaches, scientists […]. Some make use of the immune system to act and belong to a class of treatment called “immunotherapies.” ” But how do these antibodies function within the tumor? And how can we hope to improve their efficacy?

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In-Vivo Antibody Scientist Allergies 41

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.

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Vaccine Vaccination In-Vivo Genetics 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.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 40

The Pharma Data

DECEMBER 9, 2020

The new company, created by Hillhouse Capital Venture Partners, launched this week with a mission to pursue the most promising advances in medicine for underserved patients in Asia and around the world. A solution could be at hand with Overland Pharmaceuticals. Below is a roundup of some of this week’s interesting news.

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In-Vivo In-Vitro Antibody Development 52

Roots Analysis

AUGUST 23, 2024

In recent years, mRNA technology has rocked the world of medicine, making it possible to prevent and treat a vast array of diseases and disorders. Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes.

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Protein Vaccine Vaccination Immune Response 40

The Pharma Data

DECEMBER 13, 2020

She received a master’s degree in Cardiovascular Pharmacology from West China Medical Center of Sichuan University and a bachelor’s degree in Clinical Medicine from Soochow University. Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Source link.

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Business Development Development Gene Editing In-Vivo 40

XTalks

AUGUST 5, 2020

Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. Recent advances in the technology have allowed for the interrogation of distinct subsets of cell populations within tissues, and associated molecular markers that may function as important disease drivers.

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Gene Expression Development RNA Drugs 112

XTalks

AUGUST 17, 2020

This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into 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