Drug Discovery World

JANUARY 5, 2024

AbbVie and Umoja Biopharma have entered into two exclusive option and license agreements to develop multiple in-situ generated CAR-T cell therapy candidates in oncology. The first agreement provides AbbVie an exclusive option to license Umoja’s CD19 directed in-situ generated CAR-T cell therapy candidates.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Pharmaceutical Technology

MARCH 24, 2023

Under the terms of the deal, Moderna has purchased an option to license ctLNP and closed-ended DNA (ceDNA) technology from Generation Bio. Moderna will have an additional option to license a third programme targeting either immune cells or the liver.

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Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. News round-up for 19-23 February by DDW Digital Content Editor Diana Spencer.

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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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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

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

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Gene Editing Gene In-Vivo Clinical Trials 53

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Gene Therapy Gene Manufacturing Medicine 52

pharmaphorum

JULY 5, 2022

The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.

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Delveinsight

JANUARY 12, 2021

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.

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Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

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Gene Editing In-Vivo DNA In-Vitro 105

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion.

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In-Vivo Marketing Engineer Genetics 52

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

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Drug Discovery World

JULY 9, 2024

Licensing: facilitating straightforward access to genetically engineered models Genetically engineered models (GEMs) are mice and rats that have been genetically engineered to include or exclude specific DNA modifications. Therefore, licensing agreements play a crucial role in determining the availability of these models.

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In-Vivo Genetic Engineering Genetics Drugs 59

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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In-Vivo In-Vitro Antibody Development 52

The Pharma Data

APRIL 8, 2022

Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases. Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale. a Wyss Technology Development Fellow at the Institute. During his Ph.D.

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Immune Response In-Vivo Genetic Analysis Research 52

Pharmaceutical Technology

SEPTEMBER 19, 2022

The regulatory agency carried out the review of the Biologics License Application (BLA) for the therapy under the Priority Review approach. 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.

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The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

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Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

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Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

OCTOBER 2, 2023

We were able share new data that describes an expansion process in Quantum Flex that produced high quality Tscm cells in a relatively short period. With fully closed and automated devices, therapy manufacturing will hopefully be able to break the next barrier in patient access to generate safe products in record time.

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Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is an RNA editing oligonucleotide delivered to liver cells using clinically validated lipid nanoparticle (LNP) technology licensed from Genevant. Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic.

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RNA In-Vivo Genotype Genetics 52

Drug Discovery World

NOVEMBER 9, 2023

Peter O’Callaghan, PhD, Head of Expression System Sciences, Biologics and Licensing, Lonza, on: ‘Solve bispecific heavy-light chain mispairing with bYlok technology’. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

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The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. He is currently a professor at the University of Copenhagen where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery and administration of biopharmaceuticals.

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Drug Discovery World

FEBRUARY 26, 2024

For example, Vertex Pharmaceuticals’ stem-cell treatment for sickle-cell disease and transfusion-dependent β-thalassemia was authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) in November 2023, making it the first licensed therapy that uses the gene-editing tool CRISPR to help treat patients.

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Manufacturing In-Vivo Development Production 52

The Pharma Data

JULY 8, 2021

Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Amgen (NASDAQ:AMGN) announced that the U.S. Tezepelumab is being developed by Amgen in collaboration with AstraZeneca. Reese, M.D., About Severe Asthma.

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Allergies Trials In-Vivo Medicine 52

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

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Sales Vaccination Vaccine Medicine 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

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