Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

Drug Discovery World

JANUARY 27, 2023

Key features of the Winter issue include an overview of the drug discovery landscape in 2023, sector successes and challenges in the Scandinavian market, and how a drug sourced from the aerospace industry could offer treatments for multiple disease targets.

Gene Therapy 98

Gene Therapy Gene Containment Protein 98

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.

Production 52

Production Gene Protein Vaccination 52

Pharmaceutical Technology

DECEMBER 12, 2022

It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma. The development, clinical trial and marketing expenses for the cell therapy will be shared by the companies while co-commercialising the product and equally splitting profits from the US region.

Development 264

Development Gene Therapy Clinical Trials Clinical Trials 264

The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

Pharmaceutical Technology

JANUARY 31, 2023

It contains a single mRNA sequence encoding for a stabilised prefusion F glycoprotein. With this designation, we look forward to productive conversations with the FDA in the hopes of bringing our RSV vaccine candidate for older adults to the market safely and quickly.

Vaccination 182

Vaccination Vaccine Gene Therapy Gene 182

Pharmaceutical Technology

AUGUST 31, 2022

Forecasting, planning, procurement, stock levels, and marketing strategies are some of the areas that managers look at to overcome challenges with biopharmaceutical supply chains, and new innovations in the industry are revolutionising production methods.

Packaging 130

Packaging Packaging Production Containment 130

Delveinsight

FEBRUARY 22, 2021

Erythropoietic protoporphyria is a rare inherited metabolic disorder that is a result of a deficiency or inappropriate functioning of the enzyme ferrochelatase (FECH) owing to mutations in the FECH gene. Some patients with EPP often report another mutation in the ALAS2 gene, and this condition is referred to as X?linked

Marketing 52

Marketing Research Cosmetics Gene 52

Pfizer

SEPTEMBER 25, 2022

Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment. Pfizer and BioNTech Receive Positive CHMP Opinion for Conversion of COMIRNATY® Conditional Marketing Authorization to Full Marketing Authorization in the European Union. Medicines, Research.

Gene Therapy 89

Gene Therapy Containment Medicine Gene 89

Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. Given the numerous benefits of mRNA therapeutics and the increased demand for vaccines as a result of the recent COVID-19 pandemic, we believe that this market will experience significant growth in the near future.

Vaccination 52

Vaccination Vaccine DNA Protein 52

Pharmaceutical Technology

JULY 29, 2022

Once new vaccines for the Omicron variant are approved, they will partly make up for the waning interest in older Covid boosters in developed markets. And we are very prepared and have data that will answer those questions, what would be the advantage of shifting to an Omicron-containing vaccine, whether it's monovalent or bivalent.”

Manufacturing 130

Manufacturing Vaccination Vaccine Packaging 130

BioSpace

JUNE 9, 2022

Following a marathon session of the FDA's Cell, Tissue and Gene Therapies Advisory Committee, bluebird bio passed its first critical hurdle in approval for the lentiviral vector gene therapy, eli-cel.

Gene Therapy 40

Gene Therapy Gene Marketing Containment 40

Drug Discovery World

FEBRUARY 21, 2023

Kristina Tatiossian , PhD, founder of Arkitekt Agency, looks at how biotech companies are currently using social media and provides a five-step guide for how to make the most of this ubiquitous marketing tool. Furthermore, marketing teams can tailor the organisation’s following to feature specific demographics.

Marketing 52

Marketing Branding Gene Therapy DNA 52

Drug Discovery World

FEBRUARY 1, 2024

This paid-for advertorial by Twist Bioscience appeared in the Therapeutic Antibodies Guide – DDW Volume 25 – Issue 1, Winter 2023/2024 Improving gene synthesis speed can reduce workflow times, saving you invaluable time, says Twist Bioscience. But speedy gene synthesis is valuable well beyond antibody development.

Antibody 64

Antibody Gene Protein Manufacturing 64

The Pharma Data

OCTOBER 26, 2020

DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”.

Gene Therapy 52

Gene Therapy Gene Drugs Production 52

Pharmaceutical Technology

OCTOBER 18, 2022

Moderna has entered an agreement with Gavi, the Vaccine Alliance, with regard to the delivery of Covid-19 vaccines to lower-income countries supported by the Gavi COVAX Advance Market Commitment (AMC). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Vaccination Vaccine Gene Therapy Containment 130

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. coli cell bank that contains the plasmid.

DNA 52

DNA Manufacturing Gene Therapy Gene 52

The Pharma Data

JANUARY 18, 2021

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. About Taysha Gene Therapies. Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.

Gene Therapy 52

Gene Therapy Gene Drugs Production 52

Pharmaceutical Technology

SEPTEMBER 14, 2022

Successful TIL therapy depends on the infusion product containing tumour-reactive T cells that, on infusion, generate an anti-tumour immune response that causes disease regression. Synthetic gene libraries would then be used to generate reporter cells expressing neoantigens, and reporter T cells expressing potential neoantigen reactive TCRs.

Immune Response 246

Immune Response In-Vivo Production Manufacturing 246

Drug Discovery World

NOVEMBER 14, 2022

AAV vectors for diabetes gene therapy. A collaboration between PerkinElmer’s SIRION Biotech business and the Centre for Genomic Regulation (CRG) agreed to jointly develop new generation adeno-associated virus (AAV) vectors for type 1 and type 2 diabetes gene therapy in the pancreas. . A step closer to preventing type 1 diabetes.

Development 52

Development Gene Therapy Drugs Gene 52

Pharmaceutical Technology

APRIL 24, 2023

Biologics are costly to produce and sell, and the more recent modalities, such as cell and gene therapies, can be difficult to manufacture. With new drugs on the market, companies are now looking for ways to manufacture recently approved biologics, and countries are racing to claim their corner – or maintain their decades-old market share.

Contract Manufacturing 130

Contract Manufacturing Manufacturing Protein Sales 130

Roots Analysis

MARCH 1, 2023

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. It detects the potential long-term effects of the compounds which are marketed without knowing their ability to affect human and environmental health.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

Drug Discovery World

NOVEMBER 29, 2022

Now we have succeeded in designing our own DNA that contains the exact instructions to control the quantity of a specific protein,” said Aleksej Zelezniak. . The AI designs synthetic DNA, where it is easy to modify its regulatory information in the desired direction of gene expression. .

DNA 52

DNA Development Research Drugs 52

Drug Discovery World

AUGUST 10, 2022

By Mark White, Associate Director of Biopharma Product Marketing at Bio-Rad and Marwan Alsarraj, Biopharma Segment Manager, Digital Biology Group. Exactly three decades later, the first CAR T cell therapy hit the market. If the gene does not integrate into the genome, the cell will not become a CAR T cell.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

The Pharma Data

NOVEMBER 24, 2020

BRCA 1/2 or ATM gene mutation must be confirmed before Lynparza treatment is initiated. 2 Germline and somatic mutations in HRR genes, which include BRCA1/2 and ATM, are associated with potentially more aggressive prostate cancers and poor prognosis, demonstrating the importance of biomarker testing. MISSISSAUGA, ON , Nov.

Gene 52

Gene Hormones DNA Medicine 52

Roots Analysis

SEPTEMBER 18, 2023

Due to the infrastructure available for processing, freezing and storing of these container systems, blood bags are the preferred container for cell therapy biopreservation. The above figure presents a general overview of the cryopreservation process.

Engineer 52

Engineer Containment In-Vivo Production 52

pharmaphorum

JANUARY 4, 2023

The cell and gene therapy (CGT) industry is poised to achieved a significant milestone at the end of 2022: the first approval of an allogeneic T-cell therapy in the world. . However, emerging markets and remote locations, in particular, may not have the specialist facilities to maintain cryogenic storage and transport.

Gene Therapy 90

Gene Therapy Manufacturing Gene Production 90

The Pharma Data

OCTOBER 29, 2020

Co-developed with Agilent Technologies (NYSE:A), Magnis BR is a key component of Burning Rock’s strategy of empowering hospitals to run NGS tests in-house with minimized lab space and staff requirement, offering both tissue-based and ctDNA-based test kits ranging from 4-gene to 520-gene panels. About Burning Rock.

Containment 52

Containment Gene Marketing Development 52

XTalks

MAY 15, 2023

This condition arises from mutations in the GAL gene at Xq22, leading to the accumulation of glycosphingolipids within lysosomes in various tissues and organs, such as the heart, kidneys, eyes, vascular endothelium, nervous system and skin. Genetic confirmatory testing is then conducted to identify the specific mutations in the GLA gene.

FDA Approval 98

FDA Approval Gene Genetics Clinical Trials 98

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

NOVEMBER 2, 2020

In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. www.sensorion-pharma.com. www.sensorion-pharma.com. Disclaimer.

Containment 40

Containment Gene Therapy Gene Regulation 40

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.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

JANUARY 9, 2024

Thirteen new-to-market therapeutics and drugs poised to launch in 2024 will achieve ‘blockbuster’ status by 2029 or deliver game-changing benefits to patients. For patients reluctant to receive novel therapies, such as mAbs or gene therapy, efanesoctocog alfa will likely be an appealing option.

Drugs 69

Drugs Gene Editing Antibody Medicine 69

Roots Analysis

FEBRUARY 1, 2024

In the absence of suitable temperature controlled packaging solutions, the products packaged, such as vaccines, biologics, cell and gene therapies might get exposed to extreme ambient temperatures. These containers are available in a single use, as well as reusable format.

Packaging 40

Packaging Packaging Containment Production 40

XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This makes Qalsody the first approved treatment to target a genetic cause of ALS.

FDA Approval 89

FDA Approval Gene Genetics Protein 89