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. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 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. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

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. .

Gene Therapy 52

Gene Therapy Gene Development In-Vivo 52

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

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Gene Therapy Gene Manufacturing Medicine 52

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.

Licensing 52

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

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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Drugs Vaccination Vaccine Medicine 59

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 104

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

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.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

The Pharma Data

APRIL 8, 2022

They also identified several drugs that reduced the production of inflammatory cytokines in infected Alveolus Chips, which could be useful in treating excessive inflammation in the lung. Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases.

Immune Response 52

Immune Response In-Vivo Genetic Analysis Research 52

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. The treatment is indicated for usage in boys aged four to 17 years with CALD. thalassemia.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We Novartis today announced that the U.S.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

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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In-Vivo Manufacturing Production Gene Therapy 105

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.

Engineer 52

Engineer Antibody Protein In-Vivo 52

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.

RNA 52

RNA In-Vivo Genotype Genetics 52

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.

Manufacturing 52

Manufacturing In-Vivo Development Production 52

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.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. to £104.7m

Drugs 59

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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. 1) The Prescription Drug User Fee Act goal date for a decision by the FDA is during the first quarter of 2022. Amgen (NASDAQ:AMGN) announced that the U.S. Reese, M.D.,

Allergies 52

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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