Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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Gene Editing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

FEBRUARY 23, 2023

GenScript ProBio has announced a strategic collaboration with RVAC Medicines to manufacture GMP-grade plasmid DNA (pDNA) for the latter’s RVM-V001, an mRNA Covid-19 vaccine candidate. The company provides end-to-end contract development and manufacturing organisation (CDMO) services from the discovery to the commercialisation of drugs.

Vaccine 289

Vaccine Vaccination Gene Therapy Manufacturing 289

Bio Pharma Dive

NOVEMBER 14, 2022

The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

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Gene Editing RNA DNA Gene 161

Medical Xpress

NOVEMBER 17, 2022

BRCA1 (BReast CAncer Gene 1), a key gene that becomes faulty in some instances, leading to breast and ovarian cancer, plays an important role in the body's DNA repair mechanisms. BRCA1, once mutated, can cause cancer to develop.

DNA 111

DNA Production Gene Development 111

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. The exact mechanism depends on the disease in question.

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Gene Editing Gene Gene Therapy In-Vivo 145

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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Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

JULY 20, 2022

Furthermore, researchers found that one of the genes turned on by these BET proteins is ACE2, the same protein that the virus depends on to get into cells. In the latest study, Gladstone graduate student Irene Chen and the team found that the BET proteins switch on genes that block viruses, in SARS-CoV-2 infected cells.

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Protein Scientist Gene DNA 278

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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Gene Expression Regulation Gene DNA 97

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. The future of cell and gene therapies.

Gene Therapy 130

Gene Therapy Gene Marketing Manufacturing 130

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

Medical Xpress

FEBRUARY 22, 2023

Mount Sinai researchers have published a study in Alzheimer's & Dementia: The Journal of the Alzheimer's Association that sheds new light on the role of DNA methylation in Alzheimer's disease (AD).

DNA 97

DNA Research Protein Gene 97

Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

Genomics 130

Genomics Genome Medicine Development 130

Fierce Pharma

JUNE 3, 2024

subsidiary Bionova Scientific is launching a new business line to provide services around plasmid DNA (pDNA), the companies announced Monday. As part of the pDNA pivot, Bionova is setting up a new facility in The Woodlands, Texas.

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DNA Gene Therapy Contract Manufacturing Gene 80

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

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Gene Editing DNA Gene Development 58

STAT News

OCTOBER 5, 2022

Scientists rely on gene synthesis technologies as a research tool for everything from basic research to vaccine development and drug target identification. Ever since the inception of gene synthesis, there have been concerns about possible misuse of synthetic genes.

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Gene DNA Engineer Scientist 98

BioPharma Reporter

JANUARY 17, 2022

Andelyn Biosciences says the FDA's acceptance of its GMP plasmid DNA drug master file (DMF) enables the CDMO to vertically integrate its clientsâ manufacturing process, condensing timelines for developers to begin manufacturing to just three months.

Gene Therapy 103

Gene Therapy Development DNA Gene 103

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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Gene Gene Therapy Genetics DNA 262

Pharmaceutical Technology

DECEMBER 8, 2022

Touchlight and Odimma Therapeutics have entered an agreement for the development and supply of clinical material for usage in the latter’s neoantigen programme for cancer. This will back and strengthen their immunotherapy cancer treatment candidate’s development. The programme is anticipated to commence clinical enrolment next year.

DNA 147

DNA Genetics Manufacturing Production 147

pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

Gene Therapy 105

Gene Therapy Gene DNA Development 105

Scienmag

MAY 3, 2021

Detection innovation could enhance drug development and cancer research Credit: Richard Hooley/UCR Small changes in the structure of DNA have been implicated in breast cancer and other diseases, but they’ve been extremely difficult to detect — until now.

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DNA Development Drugs Research 98

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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Gene Therapy Gene Clinical Trials Clinical Trials 130

STAT News

SEPTEMBER 20, 2022

A new CRISPR startup — backed by some big names in venture capital — is planning to develop gene-editing treatments that can insert a genetic sequence of any length, at any location in the DNA strand, according to industry insiders and documents.

DNA 98

DNA Gene Editing Genome Genomics 98

pharmaphorum

NOVEMBER 22, 2021

The 2nd annual Mitochondria-Targeted Drug Development Summit returns as the only industry-led meeting focused on overcoming the challenge of treating unmet medical needs caused by mitochondrial dysfunction. The Use of Gene Therapy, Editing Technology & Innovation.

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Development DNA Drugs Gene Therapy 98

STAT News

FEBRUARY 7, 2023

From the color of our eyes to our odds of developing cancer, we’re all shaped by the genetic legacy of our ancestors. Both these modifications and their metabolic effects were shown to have passed down for at least three to six generations — something scientists once assumed was impossible.

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Gene DNA Genetics Scientist 105

Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. The collaboration aims to expand each company’s platform application through the development of new nucleic acid therapeutics, and to expedite their respective non-viral genetic medicines pipelines.

Genetics 130

Genetics Medicine Development In-Vivo 130

STAT News

NOVEMBER 14, 2022

Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA.    On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.

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Gene Editing RNA Gene Drugs 98

Medical Xpress

MAY 10, 2023

How colorectal cancer develops is not well understood, but a team led by researchers at Baylor College of Medicine reports in the Journal of Experimental & Clinical Cancer Research that silencing the gene p16, even though the DNA itself does not change, can drive colorectal cancer progression in animal models.

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Gene DNA Medicine Research 97

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genomics 244

Genomics Genome Medicine Gene Therapy 244

pharmaphorum

NOVEMBER 20, 2020

Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study. The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida.

Gene Therapy 98

Gene Therapy Gene Trials DNA 98

pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6 billion-plus.

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Gene Therapy Gene Gene Sequencing Immune Response 105

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company has developed various assets based on lentivirus vectors.

Gene Therapy 246

Gene Therapy DNA Gene Genetics 246

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: IgG gene expressing animal models. To obtain human antibody responses within a mouse, mice are genetically engineered to be humanised for their Ig genes.

Gene Expression 130

Gene Expression Gene Genetic Engineering Antibody 130

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.

Gene Therapy 105

Gene Therapy Gene Genomics Genome 105

Scienmag

JANUARY 11, 2022

Enhancers are regions of DNA that do not code for proteins, but control how genes are expressed. Super-enhancers are clusters of enhancers that together regulate genes with important roles in cell identity. Jude Children’s Research Hospital studied the Vsx2 super-enhancer and its role in the development of the retina.

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Development DNA Gene Protein 80

Pharma Mirror

FEBRUARY 23, 2021

Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility. SOMERSET, N.J. SOMERSET, N.J. Together, the addition of pDNA technology and production capabilities.

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Genetics Manufacturing Gene Therapy DNA 130