Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment.

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In-Vivo Gene Therapy Gene RNA 246

Pharmaceutical Technology

APRIL 14, 2023

Ginkgo Bioworks (Ginkgo) and the Wisconsin Alumni Research Foundation (WARF) have collaborated to discover next-generation GD2 CAR T-cell therapies to treat solid tumours. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models.

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In-Vivo Gene Therapy In-Vitro Engineer 264

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. g/dL and 45.4%

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Gene Therapy Drugs Gene Gene Editing 264

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The applications include requests for priority review. It is being assessed for the treatment of SCD or TDT patients.

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Gene Therapy Gene Editing In-Vivo Gene 246

Pharmaceutical Technology

SEPTEMBER 19, 2022

A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient. The treatment is indicated for usage in boys aged four to 17 years with CALD. 1) in Skysona-treated and untreated subjects.

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FDA Approval In-Vivo Gene Therapy Clinical Trials 246

BioSpace

JUNE 21, 2023

Elevidys, authorized Thursday to treat ambulatory patients 4 to 5 years of age, is the first in vivo gene therapy to win the FDA’s accelerated approval. It is also the first such therapy for DMD.

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Gene Therapy Gene FDA Approval In-Vivo 72

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.

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Gene Silencing In-Vivo Genetics Gene Therapy 93

BioPharma Reporter

MAY 13, 2024

Circio, a biotech firm specializing in circular RNA-based gene therapy, has unveiled two posters showcasing in vivo proof-of-concept for its circVec platform at ASGCT 2024.

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Gene Therapy Gene In-Vivo RNA 59

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 189

BioSpace

NOVEMBER 18, 2021

Avrobio’s ex vivo lentiviral gene therapy platform led to durable responses measured in years, and significant reductions in disease-related biomarkers that far exceed today’s standard of care.

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In-Vivo Gene Therapy Gene Trials 98

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.

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Genetics Medicine In-Vivo Gene Therapy 130

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.”

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Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

MAY 12, 2023

The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs). The latter partnership was recently expanded to focus on in vitro and in vivo delivery of mRNA after BioNTech pulled out of the deal with Matinas.

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Vaccination Vaccine In-Vivo In-Vitro 130

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene In-Vivo Life Science 130

Pharmaceutical Technology

MAY 10, 2023

The research teams will examine the use of CytoMed’s allogeneic gdTc on multiple AML and breast cancer subtypes in vitro and in vivo. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The study aims to use AML cells and breast cancer cell lines from patients for evaluation.

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Gene Therapy In-Vivo In-Vitro Gene 147

The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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Gene Therapy Gene Development In-Vivo 52

Pharmaceutical Technology

AUGUST 17, 2022

These programmes will include therapies and vaccines in infectious disease and oncology areas. 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. .

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RNA Vaccination Vaccine In-Vivo 147

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.

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Gene Therapy Gene Gene Editing In-Vivo 59

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

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Gene Editing In-Vivo Gene Sequencing Gene 97

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). Blood concentrations of Lp(a) are determined almost entirely by inheritance.

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Gene Editing Gene Gene Therapy In-Vivo 130

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

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

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. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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Gene Therapy Gene In-Vivo Genetics 40

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.

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In-Vivo Gene Editing Gene Therapy Gene 52

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient.

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Gene Therapy Gene Immune Response In-Vivo 52

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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Gene Editing Life Science Gene Gene Therapy 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.

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Gene Editing Genetic Disease Gene In-Vivo 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. .

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Gene Editing Gene Gene Therapy Drugs 59

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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Genome Genomics Gene In-Vivo 40

Roots Analysis

JANUARY 27, 2025

The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. The major steps involved in cell therapy manufacturing have been briefly outlined below.

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Manufacturing Contamination Production Gene Therapy 40

Pharma Marketing Network

DECEMBER 21, 2020

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. Genetic literacy among clinicians may not be as high as you think.

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Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

NOVEMBER 4, 2022

” At Novartis, she was involved with projects including multiple sclerosis therapy Kesimpta (ofatumumab), migraine therapy Aimovig (erenumab), and Parkinson’s disease, and the acquisition by Novartis of optogenetic gene therapy company Vedere Bio. .

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In-Vivo Gene Therapy Doctors Doctor 59

pharmaphorum

APRIL 29, 2021

To achieve this, the agenda will allow delegates to: Explore the translation of BBB-penetrating antibodies to non-human primates , and new possibilities for in vivo brain imaging of antibody neuropharamacokinetics. A Meeting Point for the Field’s Experts. 3-day Conference | June 28-30, 2021. About Hanson Wade.

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Drug Delivery Gene Therapy In-Vivo Antibody 52

The Pharma Data

NOVEMBER 22, 2020

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years’ experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. MILAN, Italy and NEW YORK, Nov. About Genenta Science.

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Gene Therapy Gene In-Vivo Clinical Trials 40

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.

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Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

DECEMBER 20, 2023

In 2024, pharma companies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

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Life Science Gene Editing Development Clinical Trials 119