XTalks

AUGUST 29, 2022

CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. This technology has powerful implications for therapeutic uses, such as replacing mutated or disease-causing genes or increasing the activity of cancer-fighting cells.

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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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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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pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

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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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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.

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XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

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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.

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The Pharma Data

NOVEMBER 15, 2020

a biotechnology company leveraging insights from the human genetics of repeat expansion disorders (REDs), today announced that Nessan Bermingham, Ph.D., 16, 2020 11:00 UTC. CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– Triplet Therapeutics, Inc. , A webcast of the presentation can be viewed on the conference website.

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

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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. The technology provides a way of producing hypoimmunogenic stem cells that can be differentiated into any cell type.

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Roots Analysis

FEBRUARY 26, 2024

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. It is possible that the legend of Big Foot may be based on an unfortunate person suffering from elephantiasis or gigantism or some other genetic predisposition that would have rendered him abnormal.

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Medicine Genetics Genetic Engineering Genetic Disease 52

The Pharma Data

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We It’s the cause of the mutation, not the name of the disease, that matters.”. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

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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.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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DNA Gene Gene Silencing Genome 52

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.

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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.

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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.

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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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Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.

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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. The global market for cell and gene therapies , valued at $5.9

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