BioSpace

JUNE 16, 2024

The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.

Gene Silencing 94

Gene Silencing In-Vivo Genetics Gene Therapy 94

Medical Xpress

MAY 16, 2023

They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material. That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo.

In-Vitro 90

In-Vitro In-Vivo Genetic Disease Genetics 90

STAT News

SEPTEMBER 16, 2022

Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells. Participants in the high-dose group have not yet completed the 16-week observation period.

DNA 142

DNA Gene Editing In-Vivo Genetics 142

Worldwide Clinical Trials

NOVEMBER 27, 2023

Due to inherited genetic mutations, the red blood cells of patients with severe sickle cell disease become misshaped and jagged, leading to obstructed blood vessels, intense pain, damaged organs, and other serious complications such as stroke. In June 2023, the U.S.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

Pharmaceutical Technology

OCTOBER 5, 2022

Autologous CAR-T therapy does not easily lend itself to treating CLL, as it relies on extracting functional T-cells from the patient’s blood and genetically modifying them in vitro. Autologous CAR-T agents are now becoming the standard of care for patients in the third line and later settings, providing lucrative returns for these agents.

In-Vitro 279

In-Vitro In-Vivo Sales Clinical Trials 279

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

BioTech 365

MAY 11, 2021

New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting … Continue reading →

In-Vivo 40

In-Vivo Genome Genomics Genetics 40

Scienmag

JULY 18, 2021

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

In-Vivo 89

In-Vivo DNA Genetics Medicine 89

pharmaphorum

JULY 5, 2022

The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.

In-Vivo 110

In-Vivo Bioinformatics Genetics Licensing 110

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).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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 →

Gene Editing 40

Gene Editing In-Vivo Genetics Gene 40

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.

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

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.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

NOVEMBER 4, 2022

The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. at closing.

In-Vivo 130

In-Vivo In-Vitro Genetics Drugs 130

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?

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Pharmaceutical Technology

SEPTEMBER 29, 2022

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies.

Development 130

Development In-Vivo Genomics Genome 130

World of DTC Marketing

JANUARY 31, 2021

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. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. In pharma R&D returns have declined to 1.8

In-Vivo 187

In-Vivo Generic Drugs Drugs Gene Therapy 187

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.

Gene Therapy 98

Gene Therapy In-Vivo Gene Research 98

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 →

Genetics 40

Genetics In-Vivo Engineer Medicine 40

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

MAY 18, 2023

Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We

Gene Therapy 130

Gene Therapy In-Vivo Gene Genetics 130

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing 52

Gene Editing Genetic Disease Gene In-Vivo 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.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 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

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.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

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

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.

In-Vivo 68

In-Vivo Research Gene Genetics 68

The Pharma Data

APRIL 9, 2021

Sanofi today acquired Tidal Therapeutics, a privately owned, pre-clinical stage biotech company with a novel mRNA-based approach for in vivo reprogramming of immune cells. Tidal Therapeutics utilizes a novel mRNA-based approach to in vivo reprogramming of immune cells. About Tidal Therapeutics.

In-Vivo 52

In-Vivo Research Genetics Development 52

pharmaphorum

APRIL 25, 2022

The UNC and NCSU team, led by biomedical engineer Yevgeny Brudno, report in Nature Biotechnology how they have developed an implant that can generate the CAR-T cells in vivo. In some cases where the cancer is particularly aggressive, that timeframe may simply be too long to be a viable option for the patient.

Engineer 105

Engineer In-Vivo FDA Approval Manufacturing 105

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.

In-Vivo 98

In-Vivo Research Genetics Drugs 98

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.

Life Science 117

Life Science Gene Editing Development Clinical Trials 117

XTalks

NOVEMBER 29, 2024

Poseida’s genetic engineering platforms stand out for their precision, safety and potential to open new therapeutic avenues. This CD19-directed therapy generates CAR-T cells directly within the patient’s body, addressing the scalability challenges of traditional ex vivo approaches.

Gene Therapy 74

Gene Therapy Gene Gene Editing Genetic Engineering 74

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 […].

In-Vivo 41

In-Vivo Genetics Drugs Research 41

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

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

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.

In-Vivo 52

In-Vivo Genomics Genome Gene 52