Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We feel this reflects the strong support for our vision and confirms the potential of Eligo to create a novel class of transformative genetic medicines.”

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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

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.

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

Drug Discovery World

AUGUST 7, 2023

The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Susmita Sahoo, Associate Professor of Medicine, Cardiology at Icahn Mount Sinai has been exploring the use of exosomes in gene therapy for several years.

Gene Therapy 52

Gene Therapy Gene Research In-Vivo 52

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. 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.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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

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

Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

Gene Therapy 52

Gene Therapy RNA Engineer Gene 52

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. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

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

JANUARY 5, 2024

In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation. In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation.

In-Vivo 52

In-Vivo Development Licensing Licensing 52

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

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

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. Our focus is now on accelerating progress to help realise the potential for durable and curative cell and gene therapies. 18, 2021 09:00 UTC. A total of $15.4

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

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

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

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. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

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

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

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 Genome Genomics 130

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. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

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. . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

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

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

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 Genome Genomics Gene 52

Delveinsight

FEBRUARY 11, 2021

Avrobio gene therapy eradicates toxic substrate in Fabry patient. 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. Enhancing the response to immuno-oncology in pancreatic cancer.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

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

Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

Drug Discovery World

JUNE 30, 2023

This pairing includes reprogrammed cells for both wild and mutation-specific Huntington’s Disease (H), Amyotrophic Lateral Sclerosis (ALS), and Alzheimer’s Disease (AD) and is expanding to include custom disease-relevant mutations. Disease relevant in vitro assays improve translation in vivo, leading to clinical success.

Development 52

Development Drugs In-Vitro Gene Therapy 52

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. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site.

DNA 52

DNA In-Vivo Genetics Medicine 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

Drug Discovery World

OCTOBER 27, 2023

Animal models, Ewart explains, are limited by their genetic and physiological differences from humans. With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72