Pharmaceutical Technology

DECEMBER 14, 2022

Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan.

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Pharmaceutical Technology

NOVEMBER 16, 2022

ElevateBio has entered a partnership with Affini-T Therapeutics to progress the latter’s engineered TCR-T therapies focused on Kirsten rat sarcoma viral oncogene homolog (KRAS), a dominant oncogenic driver mutation in solid tumours. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

FEBRUARY 24, 2023

It will combine the extensive capabilities of AbbVie with the new adeno-associated virus (AAV) engineering platform and the manufacturing capability of Capsida for identifying and advancing three programmes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

APRIL 6, 2023

Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. The new capabilities and IP will be incorporated under Ginkgo’s end-to-end AAV gene therapy development platform.

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pharmaphorum

JUNE 15, 2021

When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category. . The post Biogen’s ambitions in gene therapy hit by another failed pivotal trial appeared first on.

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pharmaphorum

OCTOBER 22, 2020

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. That means replacing the defective gene with a working copy should – or correcting it in situ – could stop the disease in its tracks.

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pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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XTalks

JANUARY 16, 2025

The RMAT pathway, tailored for therapies with promising potential, played a key role in accelerating bemdaneprocels progression. Bemdaneprocel is a cell therapy that uses pluripotent stem cells, cells capable of becoming any type of cell in the body.

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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.

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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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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. This increases the expression of fetal haemoglobin.

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Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. CircAde is said to be the most advanced therapeutic concept, and leverages the company’s experience in oncolytic viruses to use engineered adenoviruses to deliver circRNA to cancer cells.

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STAT News

JULY 11, 2022

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

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Gene Gene Therapy Genetic Engineering Engineer 111

Pharmaceutical Technology

APRIL 14, 2023

The company can use its mammalian cell engineering expertise and capabilities to screen CAR T-cells, to discover and optimise next-generation therapeutic candidates for its partners. WARF and Ginkgo Bioworks also intend to partner on developing a pooled in vivo screening platform to further advance the discovery of new CAR therapies.

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Pharmaceutical Technology

DECEMBER 8, 2022

It is created by taking T cells from the patient’s blood and then engineered in the laboratory to express chimeric antigen receptors. . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content. By Cytiva Thematic.

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Pharmaceutical Technology

MARCH 29, 2023

Regeneron Pharmaceuticals has collaborated with Sonoma Biotherapeutics to discover, develop and commercialise new regulatory T cell (Treg) therapies for autoimmune diseases. The two companies will together research and develop T cell therapies for Crohn’s disease and ulcerative colitis, as well as two other undisclosed indications.

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Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP. By Cytiva Thematic.

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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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STAT News

AUGUST 18, 2022

The therapy, Zynteglo, is just the third gene therapy approved by the FDA, and the first to target a chronic blood disease. It uses an engineered virus to deliver a gene fix into the bone marrow of patients. Zynteglo will cost $2.8

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Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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BioPharma Reporter

APRIL 11, 2023

Ginkgo acquires StrideBio's adeno-associated virus (AAV) capsid discovery and engineering platform assets and as well as gaining ownership of a preclinical asset.

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Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MARCH 8, 2023

The new technologies will also overcome the manufacturing bottlenecks in delivering advanced engineered cell products. Currently, there are six CAR T cell therapies that received approval from the US Food and Drug Administration (FDA) and approximately 560 programmes in ongoing studies.

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Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Meanwhile, the pharma industry is undergoing somewhat of a transformation itself.

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Scienmag

OCTOBER 19, 2020

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases Credit: Nature Biomedical Engineering Irvine, CA – October 19, 2020 – A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice (..)

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Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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Velocity Clinical Research

OCTOBER 3, 2024

As Nick Spittal states in this Advarra press release, membership in the Gene Therapy Ready (GTR) site network “allows Velocity to start studies over a month faster and provides a meaningful credential and important validation that increases sponsors’ confidence in our specialized capabilities to conduct complex clinical research safely.”

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Scienmag

JUNE 2, 2021

Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies.

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pharmaphorum

NOVEMBER 16, 2022

This is the case for the new generation of treatments known as advanced therapies, which encompasses cell and gene therapies, as well as tissue engineering. Of the innovations that have happened recently in pharma drug development, it is cell and gene therapy that have received the most attention.

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Scienmag

FEBRUARY 4, 2022

Biomedical engineers at Duke University have demonstrated a gene therapy that helps heart muscle cells electrically activate in live mice. DURHAM, N.C.

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BioSpace

MAY 10, 2021

Biogen and Capsigen forged a strategic collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to become transformative gene therapies that treat underlying genetic causes of various central nervous system and neuromuscular disorders.

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Scienmag

JUNE 9, 2021

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

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Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. These therapies involve modifying a patient’s cells or genes to treat or prevent diseases, with the promise of long-lasting effects.

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