Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

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SCIENMAG: Medicine & Health

JUNE 20, 2023

EDMONTON — Combining a cancer-targeting virus with radiation to treat brain cancer in mice was more effective than either therapy on its own according to University of Alberta research, providing hope for new treatments that combine immunotherapy with traditional surgery, chemotherapy or radiation.

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Drug Discovery Today

DECEMBER 5, 2020

OVO Biomanufacturing, a spin-out from the University of Warwick and Coventry University, is developing digital solutions to improve the efficiency of viral vaccine and gene therapy manufacture. The technology can be applied to any virus that is grown to produce a vaccine or therapy.

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Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

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ASPET

JUNE 12, 2024

We developed self-complimentary adeno-associated virus vectors (scAAV9) expressing two splice variants of human GNAO1 Gα o isoforms 1 (G o A, GNAO1.1 ) and 2 (G o B, GNAO1.2 ). This represents the first report of successful preclinical gene therapy for GNAO1 encephalopathy applied in vivo. or scAAV9- GNAO1.2

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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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Broad Institute

AUGUST 7, 2024

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy By Allessandra DiCorato August 7, 2024 Breadcrumb Home Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy The method could accelerate the development of more effective adeno-associated viruses (AAVs).

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. By Sarah C.P.

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SCIENMAG: Medicine & Health

JUNE 21, 2023

In an earlier clinical trial, in which the Bochum researchers were also involved, patients with chronic hepatitis E virus (HEV) infection were treated with the drug sofosbuvir, which was actually developed for the treatment of hepatitis C. […] Credit: © RUB, Marquard Which viral variants circulate in the blood?

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Broad Institute

SEPTEMBER 13, 2023

To be effective, these therapies must deliver the new genes, or the gene-editing machinery, to enough of the right cells in the body to have a therapeutic effect. But researchers have struggled to design molecular vehicles that can deliver gene-based therapies safely and effectively to the right cells.

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The Pharma Data

MARCH 23, 2022

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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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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Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene. Tags: Gene editing Gene therapy David Liu Nature Biomedical Engineering.

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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. MUNICH, Germany, Jan.

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Chemical Biology and Drug Design

OCTOBER 10, 2023

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. Molecular insights of HIV Reverse transcriptase and it's inhibitors. According to the Joint United Nations Programme on HIV/AIDS (UNAIDS) data, 38.4 million

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Alta Sciences

OCTOBER 7, 2024

Latest Findings in Gene Therapy Research blussier Mon, 10/07/2024 - 15:16 HTML A Promising Future for Gene Therapy Our experts have been diligently working on groundbreaking research, including a wide range of gene therapy studies, and have compiled a selection of scientific resources featuring the latest advancements.

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SCIENMAG: Medicine & Health

FEBRUARY 23, 2024

Across the globe, more than 39 million people are living with the Human Immunodeficiency Virus (HIV), the virus that causes AIDS, including more than 1.3 The majority of those afflicted — approximately 76% — have access to antiviral therapy that allows them to live with HIV as a chronic […]

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Alta Sciences

DECEMBER 12, 2024

Get Started on Your Gene Therapy Preclinical Studies Today! With a large population of NHPs that are pre-screened for adeno-associated viruses (AAVs), were ready to get started on your studies immediately. Speak with one of our experts today to get your project started right away!

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Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?

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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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Chemical Biology and Drug Design

APRIL 18, 2023

Due to the aforementioned challenges, researchers are now focusing on discovering alternative novel safe, and economically reachable therapies for the treatment of trypanosomiasis. Trypanosoma cruzi , the etiological agent of this NTD, is mostly transmitted by triatomine vectors and comprises a range of epidemiologically significant variants.

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DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. These findings underscore the potential of mRNA to redefine cancer treatment by offering precise, patient-specific therapies.

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

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The Pharma Data

JANUARY 18, 2021

Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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The Pharma Data

NOVEMBER 1, 2020

Corlieve’s lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. Maryland , US), a leading gene therapy company. Maryland , US), a leading gene therapy company. PARIS , Nov.

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addgene Blog

MAY 30, 2023

Adeno-associated virus (AAV) is a single stranded, Parvoviridae DNA virus, packaged in a non-enveloped icosahedral capsid, that can be used to express genes of interest in cell and animal models. This post was contributed by guest blogger Didem Goz Ayturk with edits and updates from Addgenies Karen Guerin and Susanna Stroik.

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Broad Institute

DECEMBER 13, 2023

The researchers also used their method to reveal which cell types and brain regions are accessible by harmless viruses called adeno-associated viruses (AAVs) that are engineered to deliver genes, demonstrating the value of this scalable approach for neuroscience research and the development of gene therapies.

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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The Pharma Data

JANUARY 17, 2021

The companies will evaluate ViGeneron’s proprietary, intravitreally injected vgAAV vectors for delivering a novel therapeutic protein to develop a gene therapy treatment for a highly prevalent eye disease. There is significant unmet medical need for a sustained therapy to treat eye diseases. MUNICH, Germany, Jan.

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The Pharma Data

JANUARY 6, 2021

A Novel Ebola Virus VP40 Matrix Protein-Based Screening for Identification of Novel Candidate Medical Countermeasures.’ The published study results showed that OYA1 was “highly effective” in reducing the spread of Ebola Virus infection in laboratory tests with the live Ebola virus carried out by NIAID.

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