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.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.

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Drug Discovery World

JANUARY 19, 2023

This paid-for advertorial by Catalent appeared in DDW Volume 24 – Issue 1, Winter 2022/2023 One significant challenge in the manufacturing of cell and gene therapies is the production of high-quality plasmid DNA (pDNA). Figure 1: The integrated development pathway for cell and gene therapies.

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Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

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The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. About Passage Bio.

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The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

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Clinical Trials Clinical Trials Gene Therapy 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 through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

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Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Drug Discovery World

AUGUST 10, 2022

To prevent these potentially fatal side effects, the US FDA recommends that manufacturers who generate CAR T cells using retroviral or lentiviral vectors screen out batches that contain more than four transgene copies per cell 13. Meanwhile, droplets that do not contain the template strand only emit weak fluorescence.

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Drug Discovery World

FEBRUARY 21, 2023

For example, CRISPR Classroom, a biotechnology education company that discusses cutting edge advances in cell and gene therapy on TikTok boasts a following of over 60,000 people of which >79% are older than 25. Furthermore, marketing teams can tailor the organisation’s following to feature specific demographics.

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Drug Discovery World

FEBRUARY 8, 2023

Svea Cheeseman , Refeyn, explains why better efficiency is needed to advance the production of viral vectors for use in gene therapies. Adeno-associated viruses (AAVs) are a promising, widely used vector for delivering gene therapies. Data were provided by the Cell and Gene Therapy Catapult (London, UK).

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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 without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

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Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

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Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. The above figure presents a general overview of the cryopreservation process.

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The Pharma Data

DECEMBER 20, 2020

Mesoblast Limited reported that its stem-cell therapy trial in ventilator-dependent COVID-19 patients with moderate to severe ARDS filed to meet the primary endpoint of 43% of reduction in mortality after 30 days of treatment. FLT180a is an AAV gene therapy. The product is an AAV9-based gene therapy for GM1 gangliosidosis.

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Trials Gene Therapy Vaccination Vaccine 52

XTalks

AUGUST 2, 2023

It contains the antiviral medications nirmatrelvir and ritonavir. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. 5) Lynparza (Olaparib) Lynparza is a PARP inhibitor , meaning it inhibits poly ADP ribose polymerase (PARP), an enzyme involved in DNA repair.

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