Scienmag

JANUARY 13, 2021

Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth Credit: Children’s Hospital of Philadelphia Philadelphia, January 13, 2021–Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science (..)

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XTalks

FEBRUARY 25, 2025

Meanwhile, Vivet Therapeutics is advancing an investigational gene therapy candidate, VTX806, which employs an adenoassociated virus (AAV) vector engineered to reinstate CYP27A1 enzymatic activity.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

pharmaphorum

OCTOBER 19, 2022

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of the amino acid become toxic to the brain and may lead to serious neurological and neuropsychological issues.

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Drugs Bacterium Genetic Disease Sales 98

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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pharmaphorum

OCTOBER 4, 2022

It is also developing an adeno-associated virus (AAV) capsid engineering platform, dubbed ‘sAAVy’, which the company claims can optimise gene delivery for treatments in a broad range of indications and tissues.

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Gene Editing Gene Genomics Genome 64

The Pharma Data

NOVEMBER 15, 2020

About TTX-3360 TTX-3360, an antisense oligonucleotide, is the first clinical candidate developed from Triplet’s proprietary thRED Engine and the first clinical candidate with the potential to modify the course of repeat expansion disorders (REDs) by targeting the DNA Damage Response (DDR) pathway.

DNA 40

DNA Genetics Engineer Genetic Disease 40

pharmaphorum

OCTOBER 5, 2022

.” The partners are still in the discovery stage of the collaboration, and Sanofi will take full responsibility for the programme once a drug candidate to take forward into preclinical and clinical testing has been selected.

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RNA Genetics Dermatology Antibody 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.

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In-Vivo Marketing Engineer Genetics 52

XTalks

MAY 4, 2021

These results suggest that CRISPRoff would potentially only need to be given as a one-time therapy for the treatment of rare genetic diseases, immune system disorders and some forms of cancers that are caused by a faulty copy of a gene.

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DNA Gene Gene Silencing Genomics 98

The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

FDA Approval 52

FDA Approval Genetic Disease Genetics Clinical Trials 52

Roots Analysis

FEBRUARY 26, 2024

Literary and archaeological evidences suggest that diseases have plagued living organisms since the beginning of time. Whether it was a common cold or a genetic disease, prayers and sacrifices to the gods were the only hope of a remedy at such times.

Medicine 52

Medicine Genetics Genetic Engineering Genetic Disease 52

XTalks

DECEMBER 13, 2023

Bolstered by world-class R&D capabilities, a highly productive scientific platform and an established management team, Alpine is dedicated to crafting first- or best-in-class multifunctional immunotherapies through distinctive protein engineering technologies, all aimed at enhancing the quality of life for patients. million and $38.8

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The Pharma Data

DECEMBER 8, 2020

Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. The engineered cells are protected by Sigilon’s Afibromer biomaterials matrix, which shields them from immune rejection and fibrosis.

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

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Gene Gene Therapy Medicine Development 52

The Pharma Data

JULY 6, 2021

About SPK-8011 for haemophilia A Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. We currently have four programs in clinical trials.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 17, 2021

Working closely with Ionis, “We connect the need to the drug development engine. The Undiagnosed Disease Network (a consortium of tertiary care institutions for that purpose) directs a large number of patients to n-Lorem, but most come from physicians. Genetic diseases are vastly more common and more complex than we used to think.”.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

XTalks

MAY 4, 2021

These results suggest that CRISPRoff would potentially only need to be given as a one-time therapy for the treatment of rare genetic diseases, immune system disorders and some forms of cancers that are caused by a faulty copy of a gene.

DNA 52

DNA Gene Gene Silencing Genomics 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

The Pharma Data

DECEMBER 27, 2020

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. aTyr Pharma completed target enrollment in its Phase Ib/IIa trial of ATYR1923 in pulmonary sarcoidosis, a form of interstitial lung disease.

Trials 52

Trials Antibody Gene Therapy Gene 52

XTalks

OCTOBER 1, 2020

Sooter has also found success in industry, leading and bringing together the work of different teams of researchers at NeuBase to help drive the company’s goal of developing cures and treatments for genetic diseases, including Huntington’s Disease. She says, “It’s been a lot of fun; it’s a decision I’m glad I made.”.

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Scientist In-Vitro Research Life Science 52

Pharmaceutical Technology

APRIL 24, 2023

Langer’s engineering lab was instrumental in the development of these lipid nanoparticles, playing into his role in co-founding Moderna. There are still possibilities that these types of nanoparticles could be used for a whole range of nucleic acid medicines, whether mRNA, DNA, or CRISPR Cas9… for different genetic diseases.

Drug Delivery 147

Drug Delivery Vaccine Vaccination Research 147

XTalks

JANUARY 6, 2025

Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

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