Drug Target Review

MAY 22, 2024

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.

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DrugBank

JUNE 13, 2024

These microscopic particles, which are smaller than 100 nanometers, can be engineered to deliver drugs to specific targets with precision.   Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.

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Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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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 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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The Pharma Data

MARCH 9, 2022

Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Scientists found the substance stopped SARS-CoV-2, the virus that causes COVID-19, from reproducing itself and also protected infected cells when tested in human lung cells.

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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

JUNE 2, 2022

Boehringer Ingelheim is pioneering a range of versatile therapeutic platforms in order to develop innovative medicines that target the tumor directly (tumor cell-directed therapies) or that enable the immune system to target the tumor (immune cell-targeted therapies). About Boehringer Ingelheim. For ongoing news, visit www.a-star.edu.sg.

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NIH Director's Blog: Drug Development

FEBRUARY 1, 2022

The NIH continues to support the development of some very innovative therapies to control SARS-CoV-2, the coronavirus that causes COVID-19. They found in mouse models of severe COVID-19 that intravenous infusion of an engineered ACE2 decoy prevented lung damage and death. How’s that?

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The Pharma Data

APRIL 15, 2022

Designed computationally and refined in the laboratory, the new protein therapies thwarted infection by interfering with the virus’ ability to enter cells. The top protein neutralized the virus with similar or greater potency than antibody treatments with Emergency Use Authorization status from the U.S.

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The Pharma Data

JANUARY 11, 2021

a San Diego-based biotechnology company with an array of technology platforms for antibody discovery and optimization, and novel NK and T cell engager generation, today announced licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development.

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Agency IQ

NOVEMBER 3, 2023

regulatory landscape for probiotics and other microbiome-based therapies Probiotics, a type of microbiome-based therapy containing live micro-organisms, are broadly available on drug store and grocery store shelves, but the regulatory classifications for products containing the same micro-organism can differ dramatically.

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Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

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The Pharma Data

DECEMBER 31, 2020

Taysha Gene Therapies – Texas-based Taysha Gene Therapies bolstered its leadership team with the appointments of Greg Gara as senior vice president of Manufacturing and Kimberly Lee as senior vice president of Corporate Communications and Investor Relations. Source link.

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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. By Sarah C.P. However, delivering the complex gene editing machinery to cells in living animals has been challenging.

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Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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Codon

MAY 18, 2023

And the article doesn’t mention how Pairwise engineered the mustard to be “less pungent.” A gene therapy for Duchenne muscular dystrophy is (probably) on the way. ” Then — that same day — an advisory panel voted 8-6 to recommend approval for the gene therapy anyway. .” in a series A.

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Codon

MAY 18, 2023

And the article doesn’t mention how Pairwise engineered the mustard to be “less pungent.” A gene therapy for Duchenne muscular dystrophy is (probably) on the way. ” Then — that same day — an advisory panel voted 8-6 to recommend approval for the gene therapy anyway. .” in a series A.

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DNA Engineering Therapies Virus 52

PLOS: DNA Science

JANUARY 4, 2024

The end-of-year FDA approval of the first CRISPR-based therapy , for sickle cell disease, came a mere dozen years after Jennifer Doudna and Emmanuelle Charpentier introduced the technology. Unlike gene therapy as originally-envisioned since the 1950s, which adds a functioning copy of a gene, CRISPR can add, remove, or replace a DNA sequence.

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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The Pharma Data

AUGUST 16, 2021

During challenge with the original SARS-CoV-2 virus, animals vaccinated with CV2CoV were found to be better protected based on highly effective clearance of the virus in the lungs and nasal passages. Clearance of the virus in the lungs and nasal passages of the animals was tested following challenge infection with the original virus.

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The Pharma Data

DECEMBER 10, 2020

In the study, immunization with the hAd5-COVID-19 vaccine inhibited SARS-CoV-2 virus replication in 100% (10 of 10) of Rhesus macaques, with a drop in viral replication starting on the first day of vaccine administration, and undetectable viral levels as early as three to five days post-challenge in most of the animals.

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The Pharma Data

DECEMBER 2, 2020

STRASBOURG, France–( BUSINESS WIRE )– Regulatory News: Transgene SA (Paris:TNG), a biotech company that designs and develops virus-based immunotherapeutics against cancer, announced that the Board, at its meeting today, approved the appointment of Hedi Ben Brahim as the Company’s new Chairman and CEO, effective January 1 st , 2021.

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Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Codon

MAY 11, 2023

Scroll to the end for a beautiful chart about the Anthropocene & some details on insect engineering companies. It is used to prevent respiratory syncytial virus, or RSV, which causes about 60,000 hospitalizations in this age group each year. link ) Emily Whitehead was the first child to receive a CAR-T cell therapy.

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Vaccine Engineering Licensing Licensing 52

Codon

MAY 11, 2023

Scroll to the end for a beautiful chart about the Anthropocene & some details on insect engineering companies. It is used to prevent respiratory syncytial virus, or RSV, which causes about 60,000 hospitalizations in this age group each year. link ) Emily Whitehead was the first child to receive a CAR-T cell therapy.

Vaccine 52

Vaccine Engineering Licensing Licensing 52

Drug Target Review

SEPTEMBER 11, 2023

After numerous mutations of the StcE mucinase, the team developed a version known as eStcE (“engineered sticky”) that met their requirements. The path ahead While this study is a significant stride in cancer research, it hasn’t yet led to a new cancer therapy for humans.

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The Pharma Data

OCTOBER 27, 2020

At Columbia, Dr. Clynes developed several novel therapeutic approaches in cancer and autoimmunity, including DC targeting for cancer vaccines and Syk and JAK mediated inhibition of DCs and T cells in autoimmunity therapy. OSLO, Norway , Oct. Fennel cover the key scientific focus areas of Targovax. Dean A.

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Eye on FDA

MARCH 26, 2021

A meeting scheduled for May 8 of the Cellular, Tissue and Gene Therapies Advisory Committee was slated to be a hybrid event – with members of the committee participating virtually and FDA staff appearing onsite at the agency campus, but was altered to become an all-virtual event via Adobe Connect.

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The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration (FDA) to begin a Phase I clinical trial of hAd5-COVID-19, the company’s novel COVID-19 vaccine candidate that targets both the inner nucleocapsid (N), engineered to activate T cells, and outer spike (S) protein, engineered to activate antibodies against the coronavirus (SARS-CoV-2).

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The Pharma Data

APRIL 1, 2021

In 2017, Tecartus was granted Breakthrough Therapy Designation by the FDA for relapsed or refractory adult B-cell precursor ALL. If approved, Tecartus would become the first and only chimeric antigen receptor (CAR) T-cell therapy approved for adults (?18 Tecartus is an autologous, anti-CD19 CAR T cell therapy. About Tecartus.

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The Pharma Data

MARCH 14, 2023

About QDENGA ® ▼ (Dengue Tetravalent Vaccine [Live, Attenuated]) QDENGA ® (TAK-003) is a dengue vaccine that is based on a live-attenuated dengue serotype 2 virus, which provides the genetic “backbone” for all four dengue virus serotypes and is designed to protect against any of these serotypes. 8 The study is comprised of five parts.

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The Pharma Data

JANUARY 17, 2021

John is currently Chief Executive Officer and President of Assembly Biosciences, a clinical-stage biotechnology company committed to advancing novel therapeutics to improve treatment options for hepatitis B virus worldwide. ” About Evox Therapeutics.

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The Pharma Data

OCTOBER 26, 2020

Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. SAN JOSE, Calif. , AR-711 (COVID-19).

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The Pharma Data

DECEMBER 3, 2020

Today, transplant patients who do not respond or experience adverse events related to existing therapies may be at higher risk for complications from the virus, including transplant failure and death. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. About Takeda’s Maribavir Phase 2 Study.

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