BioSpace

APRIL 29, 2024

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

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Drug Discovery World

MARCH 19, 2024

AT-108 is a first-in-class, off-the-shelf gene therapy that directly reprogrammes tumour cells into antigen-presenting dendritic cells, ultimately leading to a personalised anti-tumour immune response. The post €30m raised for first-in-class in vivo cell reprogramming platform appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 20, 2024

The proceeds will be used to advance CPTX2309, Capstan’s lead in vivo chimeric antigen receptor T cell (CAR-T) candidate, to early clinical proof-of-concept in autoimmune disorders, and to further develop Capstan’s tLNP pipeline. Biotechnology company Capstan Therapeutics has raised $175 million in an oversubscribed Series B financing.

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Bio Pharma Dive

OCTOBER 3, 2023

The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.

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Drug Discovery World

FEBRUARY 22, 2024

Under the terms of the agreement, the companies plan to combine Kelonia’s iGPS with Xyphos’ ACCEL technology to develop in vivo CAR-T cell therapies targeting up to two programmes. The post Companies agree $800m deal for off-the-shelf in vivo CAR-T cell therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 14, 2023

It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

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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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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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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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Bio Pharma Dive

OCTOBER 25, 2023

The study results, while only from three participants, offer a glimpse at how one of the few in vivo CRISPR therapies in U.S. human testing is working.

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BioSpace

APRIL 24, 2024

Regeneron will use Mammoth Biosciences’ tiny Cas enzymes to deliver in vivo CRISPR-based gene editing therapies to tissues and cell types beyond the liver, the companies announced Thursday.

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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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BioTech 365

AUGUST 25, 2021

Precision BioSciences to Host In Vivo Gene Editing R&D Event on September 9, 2021 Precision BioSciences to Host In Vivo Gene Editing R&D Event on September 9, 2021 DURHAM, N.C.–(BUSINESS –(BUSINESS WIRE)–Precision BioSciences, Inc.

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BioTech 365

OCTOBER 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Intellia, LogicBio fuel hope for in vivo gene editing with new animal data in rare diseases: report.Intellia, LogicBio fuel hope for in vivo gene editing with new … Continue reading →

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Pharmaceutical Technology

JULY 14, 2022

Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II (MPSII) or Hunter syndrome. The company noted that this gene therapy is the fourth one to receive orphan drug designation.

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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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BioTech 365

AUGUST 31, 2021

Ixaka to present in vivo gene delivery platform for CAR-T and related bioproduction challenges at 6th CAR TCR Summit Ixaka to present in vivo gene delivery platform for CAR-T and related bioproduction challenges at 6th CAR TCR Summit CSO, Cecile … Continue reading →

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BioSpace

OCTOBER 18, 2023

The trial, which is the first late-stage study of an in vivo CRISPR treatment in the U.S., will start by the end of 2023. Intellia's NTLA-2001 is a treatment candidate for transthyretin amyloidosis cardiomyopathy.

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BioSpace

JULY 6, 2021

This appears to be a second example of CRISPR gene editing inside the body, as opposed to in cell cultures.

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Pharmaceutical Technology

OCTOBER 14, 2022

Pharmaceutical companies Xcell Biosciences (Xcellbio) and aCGT Vector have partnered to expedite the development of cell and gene therapies. The two companies will aim to improve the manufacturing and analytic procedures used to develop personalised cell and gene therapies to treat cancer patients.

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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 By focusing on the in-vivo delivery of genetic cargoes to the microbiome, Eligo’s hopes to go beyond traditional gene therapy and gene editing.

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BioPharma Reporter

JANUARY 10, 2022

have announced a strategic collaboration and option agreement for the use of Mammothâs CRISPR systems to develop in vivo gene-editing therapies. Bayer AG and Mammoth Biosciences, Inc.,

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Medical Xpress

MAY 16, 2023

They study gene variation and mutations that cause rare genetic diseases. 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. A mutation is a permanent change in the genetic material.

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pharmaphorum

JANUARY 10, 2024

Precision BioSciences licenses non-cancer uses of its allogeneic CAR-T therapy azer-cel to TG Therapeutics as it pivots to in vivo applications of its gene-editing technology

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BioSpace

SEPTEMBER 29, 2021

EDIT-101 targets a disease-causing mutation in the CEP290 gene that causes degeneration in ocular photoreceptor cells, which are critical for normal vision.

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Pharmaceutical Technology

APRIL 13, 2023

Takeda has announced that it will be pivoting away from its discovery and preclinical programmes in adeno-associated virus (AAV) gene therapies. Takeda’s announcement underlines the risk associated with gene therapy R&D at the preclinical stage and the fact that many current AAV programs are unlikely to reach late-stage trials.

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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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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.

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Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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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.

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Pharmaceutical Technology

MAY 19, 2023

MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials. “We Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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BioSpace

AUGUST 3, 2021

With this milestone, the field is now wide open for the next in vivo CRISPR success, and Editas is hoping that it will be in the ocular disease space.

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Pharmaceutical Technology

APRIL 14, 2023

All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. Topic sponsors are not involved in the creation of editorial content.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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pharmaphorum

JULY 9, 2021

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The post NICE rejects Orchard’s gene therapy for rare childhood disease MLD appeared first on.

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BioSpace

OCTOBER 18, 2021

On Monday, LogicBio Therapeutics unveiled clinical trial results demonstrating the first-ever in vivo, nuclease-free genome editing in little humans.

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