Pharmaceutical Technology

JUNE 8, 2023

The ttLNP platform can deliver a range of high-impact genomic payloads, including gene editing, siRNA, mRNA, DNA and gene-writing apparatus, to tissues, cells and organs. The development will be advanced through both internal pipeline programmes and external collaborations.

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Genome Genomics Medicine Development 130

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Knowledge of their disposition also helps in understanding the cells where the genetic material was delivered.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

APRIL 17, 2024

DS: You say on your website that your approach is applicable to any disease where increasing protein expression is beneficial. What diseases are you currently targeting? JMB: Hundreds of genetic diseases involve a missing or deficient protein and/or enzyme. Can you give some examples?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease.

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Gene Therapy Gene Genetic Disease Genetics 52

STAT News

SEPTEMBER 16, 2022

Enjoy, and see you soon … Intellia Therapeutics reported that the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells , STAT writes. Continue to STAT+ to read the full story…

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Gene Editing Genetic Disease Genetics Protein 94

Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

Genome 52

Genome Genomics Genetics Genetic Disease 52

Drug Discovery World

NOVEMBER 7, 2022

From a general healthcare perspective, access to this sort of data is invaluable, but this is particularly noteworthy in that DNA tests have historically been limited in these regions on account of cost and availability of practitioners, services and resources.

Genome 98

Genome Genomics Development Genetics 98

Pharmaceutical Technology

MAY 8, 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's lead asset involving this technology is isaralgagene civaparvovec (ST 920).

Genetics 130

Genetics DNA Genetic Engineering Antibody 130

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

Gene Therapy 135

Gene Therapy Gene Marketing Genetic Disease 135

Roots Analysis

FEBRUARY 21, 2022

Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Concluding Remarks.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

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

pharmaphorum

SEPTEMBER 6, 2021

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods. So far, subjects in Phearless have received doses of either 2e13 vg/kg or 6e13 vg/kg of BMN 307, although the protocol does include a third, higher-dose group.

Gene Therapy 82

Gene Therapy Gene Genetic Disease DNA 82

Pharma Mirror

APRIL 21, 2024

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. Messenger RNA (mRNA) is a single-stranded ribonucleic acid transcribed from the DNA chain, carrying the encoding information for protein synthesis.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

pharmaphorum

JANUARY 20, 2021

Ella, 28, was diagnosed with cystic fibrosis – a rare genetic disease which causes a build-up of thick, sticky mucus in the lungs, digestive tract, and sinuses – at 18 months old. Once inhaled into the lungs, the virus sets about binding to and inserting its own DNA into the targeted bacteria. I was really sick.

Bacteria 102

Bacteria Bacterium DNA Doctors 102

The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

DNA 52

DNA Scientist Genetic Disease Research 52

XTalks

DECEMBER 13, 2023

The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. This has tremendous potential for curing diseases that are caused by faulty/mutated genes.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

Pharmaceutical Technology

JANUARY 10, 2023

The multi-year collaboration will focus on discovering precision genetic medicines by developing a new platform for complete gene insertion by delivering a single vector of gene editing and DNA cargo. Asklepios expects that the partnership can extend the gene editing reach for lung and liver disease targets.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

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.

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 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 through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

AUGUST 29, 2022

This technology has powerful implications for therapeutic uses, such as replacing mutated or disease-causing genes or increasing the activity of cancer-fighting cells. Viral vectors have traditionally been used in gene editing where the shells of viruses carry the template DNA into cells, but this method has its disadvantages.

Gene Editing 98

Gene Editing DNA Gene Genome 98

pharmaphorum

APRIL 20, 2022

In 2012, Jennifer Doudna and Emmanuelle Charpentier published a paper in Science where they outlined isolating the components of the CRISPR-Cas9 system and demonstrated how it could be used to cut specific sites in isolated DNA. The publication and their work eventually led to the pair being awarded the Nobel Prize in Chemistry in 2020.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. We believe it can now do the same for rare diseases.” “We We have shown that mRNA holds an unprecedented therapeutic potential for incurable genetic diseases, in particular liver conditions.

RNA 52

RNA Protein Vaccination Vaccine 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.

DNA 52

DNA Gene Gene Silencing Genome 52

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. , Triplet is headquartered in Cambridge, Mass. Source link.

DNA 40

DNA Genetics Engineer Genetic Disease 40

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

The Pharma Data

AUGUST 3, 2021

The use of a sham procedure in STEER is included to provide a comparison group for an unbiased collection and assessment of efficacy, safety and tolerability of OAV-101 IT for this older population where the disease progression is slower. Spinal Muscular Atrophy (SMA) ?

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 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 Clinical Trials 52

XTalks

DECEMBER 22, 2021

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA Therapeutics. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

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.”.

Scientist 52

Scientist In-Vitro Research Life Science 52

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.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147