BioPharma Reporter

APRIL 6, 2022

The FDA has released two draft guidance documents: for human gene therapy products incorporating human genome editing, and for CAR T cell products.

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XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Drug Discovery World

MAY 1, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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BioPharma Reporter

FEBRUARY 21, 2022

PerkinElmerâs Sirion Biotech business is teaming up with the Centre for Genomic Regulation (CGR) to jointly develop new generation adeno-associated virus (AAV) vectors for type 1 and type 2 diabetes gene therapy in the pancreas.

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BioPharma Reporter

JUNE 23, 2022

BIO, the worldâs largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.

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Drug Discovery World

APRIL 24, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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pharmaphorum

SEPTEMBER 26, 2022

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Download the full event guide to find out more.

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Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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Drug Discovery World

APRIL 16, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post rAAV Genome sequencing uncovers transgene integrity and packaging impurities appeared first on Drug Discovery World (DDW). A live Q&A session follows the webinar presentation.

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BioPharma Reporter

MARCH 13, 2023

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

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Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

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Drug Discovery World

DECEMBER 5, 2022

Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. . In the study, researchers used ‘fossilised’ virus DNA sequences contained within animal genomes to reconstruct the long-term evolutionary history of parvoviruses.

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Drug Discovery World

JULY 3, 2023

Cytiva and BioCentriq, a New Jersey-based cell and gene therapy contract development and manufacturing organisation (CDMO), have been awarded funding from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), a public-private partnership whose mission is to accelerate biopharmaceutical innovation.

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XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

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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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pharmaphorum

APRIL 19, 2021

French genomic medicines firm SparingVision has agreed to buy GAMUT Therapeutics, a biotech specialising in gene therapies for inherited eye diseases such as retinitis pigmentosa (RP) that could compete against Roche/Spark’s Luxturna in a wider patient group. . million ($52.7

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Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

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Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

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BioSpace

DECEMBER 11, 2023

Backed by ARCH Venture Partners and Fujifilm, as well as technology licensed from MIT, Tome is looking to create curative cell and integrative gene therapies.

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BioTech 365

FEBRUARY 10, 2021

Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence PARIS–(BUSINESS WIRE)–WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, … Continue reading (..)

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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. million price point per dose.

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Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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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. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham. Continue to STAT+ to read the full story…

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pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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BioTech 365

OCTOBER 27, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: ElevateBio cuts a deal for Life Edit, adding genome engineering tech to support gene therapy pipeline.ElevateBio cuts a deal for Life Edit, adding genome engineering tech to … Continue reading →

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BioTech 365

MAY 31, 2021

WhiteLab Genomics and the Accelerator for Technological Research in Genomic Therapies Announce Their Collaboration on Artificial Intelligence for the Bioproduction of Gene Therapies WhiteLab Genomics and the Accelerator for Technological Research in Genomic Therapies Announce Their Collaboration on Artificial Intelligence … Continue reading → (..)

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BioSpace

NOVEMBER 20, 2023

will receive $50 million over two years from Flagship to study somatic genomics with an eye to finding new targets for gene therapies. Quotient Therapeutics, co-located in Cambridge, Mass. and Cambridge, U.K.,

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BioPharma Reporter

MAY 7, 2024

Broken String Biosciences, a genomics company driving cell and gene therapy developing, has entered a research collaboration with the Francis Crick Institute, a biomedical discovery institute dedicated to understanding the biology underlying health and disease.

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Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. The post Partners working to realise the future of genomic medicine appeared first on Drug Discovery World (DDW).

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pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Drug Discovery World

APRIL 17, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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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

JUNE 16, 2023

Benefiting from Abu Dhabi’s improved genomics capabilities, the deal seeks to expand research into genomic medicine and genetic diseases to provide patients in the UAE and beyond with improved access to new tools and treatments.

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