The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. 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.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

OCTOBER 5, 2022

Autologous CAR-T therapy does not easily lend itself to treating CLL, as it relies on extracting functional T-cells from the patient’s blood and genetically modifying them in vitro. Autologous CAR-T agents are now becoming the standard of care for patients in the third line and later settings, providing lucrative returns for these agents.

In-Vitro 279

In-Vitro In-Vivo Sales Clinical Trials 279

Pharmaceutical Technology

MARCH 9, 2023

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Roots Analysis

MARCH 1, 2023

Genotoxicity testing refers to the evaluation of detrimental effects of chemical or physical agents on the genetic processes and related hereditary material of living cells. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 52

World of DTC Marketing

JANUARY 31, 2021

Smaller biotechs that are developing new drugs usually don’t have the resources to take new drugs to market. The cost of bringing a drug to market decreased from $2,168 million in 2018 to $1,981 in 2019. Still, she is missing a lot of facts at a time when hospital and health insurance costs are soaring. percent from 2018.

In-Vivo 187

In-Vivo Generic Drugs Gene Therapy Drugs 187

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 142

Gene Editing Gene Gene Therapy In-Vivo 142

Drug Discovery World

FEBRUARY 15, 2024

The strengths of etherna’s lipid based nanoparticle (LNP) platform have been showcased in a peer-reviewed paper in Advanced Functional Materials. etherna’s Vice President, Technology & Innovation, Stefaan De Koker described the paper as a “landmark” for the company and its technology.

Immune Response 52

Immune Response In-Vivo Vaccination Vaccine 52

Drug Discovery World

MARCH 20, 2024

billion for bringing a new drug to market, a process extending over a decade. They can be developed from both normal and diseased tissue and remain stable when expanded ex vivo in the lab, offering a distinct advantage for testing off-target and on-target toxicities within the same patient sample.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 57

Gene Editing Gene In-Vivo Clinical Trials 57

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . Besides underdiagnosis, a lack of standardized treatment option forms a huge clinical unmet need.

Marketing 52

Marketing Gene Drugs Protein 52

Drug Discovery World

APRIL 12, 2023

Whilst mouse models have been standard use in clinical trials due to the animal’s genetic and physiological similarities, the issue of translatability remains. Animal models can be great indicators to assess how a drug will perform in vivo, in a particular species, but the human body is complex. This is essential.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

pharmaphorum

AUGUST 24, 2021

It will focus on ‘ex vivo’ therapies, which involves harvesting cells from patients, modifying them and then infusing then back into the body. “This new collaboration further expands our toolkit in cell and genetic therapies,” said Bastiano Sanna, Vertex’ chief of cell and genetic therapies. .

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

APRIL 12, 2021

C4XD said it has developed a molecule small enough to be formulated into a pill, which has also been able to block IL-17 in vivo. This follows a tie-up between C4XD, Sano Genetics and BenevolentAI to accelerate research into inflammatory bowel disease, which was signed in February.

Drugs 52

Drugs In-Vivo Licensing Licensing 52

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. in a statement. . Libra Therapeutics.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

Drug Discovery World

FEBRUARY 15, 2023

Genetically modified T cells stimulated with IL2 are now administered as cell therapy for multiple cancer cell types with beneficial results 3,4. Cancer is a complex heterogenous multistep disease characterised by uncontrolled cell proliferation combined with a dysregulated immune response. This continues to be the standard of care.

Research 52

Research In-Vitro In-Vivo Development 52

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Rare diseases can often be progressive, chronic and fatal. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Roots Analysis

JANUARY 14, 2024

Notably, biologics have higher success rates than conventional small-molecule drugs owing to lesser off-target toxicity of biologics, which is one of the key reasons of failures in small molecule drugs development. They are different from small molecules in terms of their size and complexity.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

FEBRUARY 6, 2023

MT: Can you provide me with an overview of what Infinimmune does currently and the potential it sees in the antibody-based therapeutics market? DDW’s Megan Thomas caught up with Wyatt McDonnell, CEO and co-founder of Infinimmune. This has consequences for safety and efficacy.

Antibody 52

Antibody Immune Response Genome Genomics 52

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. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

Engineer 52

Engineer Containment In-Vivo Production 52

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

Pharmaceutical Technology

APRIL 12, 2023

Pharmaceutical research has long relied on non-human primate models for early-stage discoveries, but their use continues to cause controversy. This was part of an investigation into an international primate smuggling ring. Globally, non-human primates make up a relatively small percentage of the animals used in research.

Regulation 245

Regulation Vaccination Vaccine Medicine 245

Drug Discovery World

JULY 22, 2022

In vivo models, particularly HIS models, allow preliminary investigation of cell-based therapeutic strategies—often in combination with other therapeutic modalities—to overcome the limitations and challenges of T cell-based immuno-oncology treatments and identify effective options for clinical investigation. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Antibody 52

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

Engineer 52

Engineer Antibody Protein In-Vivo 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. But psychopharmacology developments petered out, and few new drugs reached the market during the last four decades. to £104.7m

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Drug Discovery World

MAY 10, 2023

Due to the high costs associated with new drug development, pharmaceutical companies are under pressure to get effective drugs to market as fast as possible while ensuring they are safe for the intended patient population and dosing timeline, which can be lifelong for some drugs. billion and take over 10 years.

Bioassay 52

Bioassay Genotoxicity Drugs In-Vivo 52

Drug Discovery World

JANUARY 23, 2023

Demonstrating benefits such as fewer side effects and lower cost to enhanced efficacy and accessibility, this emerging therapeutic modality is giving hope to patients and clinicians. The momentum of RNA technologies is apparent in the significant increase in scientific knowledge, IP and deal-making value over recent years.

RNA 98

RNA Genome Genomics Engineer 98

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Already in markets in China, BeiGene’s Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc? and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

The Pharma Data

DECEMBER 27, 2020

Changes in heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) are being monitored in patients treated with LYS-SAF302 to provide evidence of in vivo biological activity of the drug and demonstrate proof of concept. Average reductions were highly statistically significant. About Lysogene. Sarepta Therapeutics, Inc.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

Drug Discovery World

SEPTEMBER 28, 2022

In fact, the market research firm, Bekryl, predicts that AI has the potential to offer over $70 billion in savings for the drug discovery process by 2028. . According to Hampleton Patners’ latest Healthtech M&A market report2, digital health companies raised a total of $57.2 The simple answer is no.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

The Pharma Data

OCTOBER 15, 2020

marketing and diagnostics. 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. The Advance awards recognize the work of remarkable global Australians making an extraordinary impact worldwide.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Drug Discovery World

JULY 5, 2023

Global Cancer Segment Market Manager at Agilent Technologies, recently sat down with DDW Editor Reece Armstrong to tell us about the developments in the ‘golden age’ of cancer research. Mark Garner, PhD., RA: Please can you tell us a bit more about your role at Agilent? MG: I am responsible for Agilent’s cancer segment.

Research 98

Research Protein Scientist Immune Response 98

Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

Manufacturing 40

Manufacturing Production Contamination Gene Therapy 40