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. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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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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Pharmaceutical Technology

SEPTEMBER 9, 2022

US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developing RNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored.

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Drug Discovery World

JUNE 29, 2023

The latest eBook by DDW ‘The impact of genomics on drug discovery: Creating new opportunities’ is out now. This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

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Drug Discovery World

JUNE 29, 2023

This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects.

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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. If you work in quality control, quality assurance, or process development – we’ve listened and we’ve answered. Download the full event guide to find out more.

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Pharmaceutical Technology

APRIL 12, 2023

FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients. Repotrectinib has been developed to combat cancer resistance to existing therapies and exert consistent anti-tumor activity.

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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. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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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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STAT News

AUGUST 17, 2022

When the Smithsonian National Museum of Natural History opened its genomics exhibit in 2013, the field was just celebrating the 10th anniversary of the completed Human Genome Project. Sequencing that first genome cost over $500 million. The genomes since cost $10,000. The development of CRISPR-Cas9 landed a Nobel Prize.

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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”.

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Drug Discovery Today

APRIL 24, 2023

Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

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Drug Discovery World

DECEMBER 29, 2023

Known as ‘CReATiNG’ (Cloning Reprogramming and Assembling Tiled Natural Genomic DNA), the method offers a simpler and more cost-effective approach to constructing synthetic chromosomes. The thing about most synthetic genomics research is that it involves building chromosomes or genomes from scratch using chemically synthesised DNA pieces.

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Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Scribe is also entitled to receive potential payments worth over $1bn on meeting development and commercial milestones.

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pharmaphorum

OCTOBER 19, 2022

million ($40 million) first-round financing that will be used to explore so-called ‘dark’ regions of the human genome. M Ventures’ Dr Bauke Anninga said that that the startup has a “differentiated platform technology has the potential to fundamentally shift the way we discover and develop precision medicines.

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Pharmaceutical Technology

OCTOBER 11, 2022

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. Ochre Bio will also expand its platforms for drug discovery and development that include a new RNA chemistry and an exploratory platform for expanding its research to more liver diseases.

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Drug Discovery Today

OCTOBER 27, 2022

In the field of life sciences, Lithuania is best known for the discovery of gene scissors by Prof. The country now hosts the EMBL Partnership Institute for Genome Editing Technologies at the Vilnius University Life Sciences Center aiming to advance gene editing technologies. Virginijus Šikšnys. One of them is the Jones!Lab,

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BioTech 365

MARCH 2, 2021

ERS Genomics Licenses CRISPR Gene Editing Technology to Otsuka Pharmaceutical ERS Genomics Licenses CRISPR Gene Editing Technology to Otsuka Pharmaceutical License to Nobel prize winning CRISPR technology supports internal research and development DUBLIN, Ireland–(BUSINESS WIRE)–ERS Genomics Limited, which was formed … Continue reading (..)

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Drug Discovery World

JUNE 21, 2023

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Exact Sciences, Guardant Health, Illumina, MSD and Novartis have come together to launch the European Coalition for Access to Comprehensive Genomic Profiling (ECGP).

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Medical Xpress

MAY 11, 2023

Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development. However, for the vast majority of those mutations, researchers are unsure of how they contribute to cancer because there's no easy way to study them in animal models.

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pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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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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Drug Discovery World

FEBRUARY 8, 2024

In 2023, 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. How is the titer determined?

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Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Scienmag

DECEMBER 20, 2021

Department of Energy (DOE) announced a plan to provide up to $30 million for basic research that will lead to transformative approaches to determine and validate gene function in plant species relevant to the sustainable production of bioenergy and bioproducts.

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Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

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Drug Discovery World

OCTOBER 26, 2022

A study by the Qatar Genome Research Consortium, led by Dr Lotfi Chouchane from Weill Cornell Medicine-Qatar, has reported the first landscape of cancer germline mutations – hereditary mutations – in the Qatari population. . These variants increase the risk of developing breast cancer in women and prostate cancer in men. .

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pharmaphorum

AUGUST 4, 2022

Unlocking the secrets of the human genome has long been an ambitious pursuit for researchers around the world. Today, the landscape of genomic testing and research is rapidly progressing, with significant scientific and technological advances driving a paradigm shift in the understanding of oncology at a molecular level.

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Scienmag

JANUARY 11, 2022

LA JOLLA—(January 11, 2022) What determines how a cell’s genome is regulated to ensure proper growth and development? Turns out, the parts of the genome that are turned on or off in each cell-type or tissue play a major role in this process. Now, a team at Salk has shown that the CLASSY gene family […].

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BioTech 365

SEPTEMBER 3, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers develop an engineered ‘mini’ CRISPR genome editing system.The common analogy for CRISPR gene editing is that it works like molecular scissors, cutting out select sections of … Continue reading → (..)

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BioTech 365

MARCH 24, 2021

Ocean Genomics Partners With Geninus to Co-Develop RNA-Based Biomarkers and Advance Research Ocean Genomics Partners With Geninus to Co-Develop RNA-Based Biomarkers and Advance Research PITTSBURGH–(BUSINESS WIRE)–#RNA–Ocean Genomics (Pittsburgh, PA), a world leader in AI-based gene expression analysis and RNA biomarker … Continue (..)

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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Drug Discovery World

AUGUST 16, 2023

Therefore, it is now more critical than ever to advance the science to develop the next generation of RNA therapies, leveraging this molecule as a therapeutic modality with transformational potential. In actuality, the protein-coding portion of our genome is comparable in identity and number with the humble fruit fly or worm.

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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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