Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

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Gene Editing Gene Gene Therapy In-Vivo 130

Drug Discovery World

MAY 17, 2024

The top stories: Gene therapy cures profound genetic deafness within 24 weeks The investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) within 24 weeks, according to trial data presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference.

Gene Therapy 52

Gene Therapy Gene Drugs In-Vivo 52

WCG Clinical

MAY 28, 2024

Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. Today there are six CAR-T products with FDA approval for the treatment of multiple myeloma and B cell malignancies, with many related and next-generation products under development.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

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.

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Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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Gene Editing In-Vivo Gene Sequencing Gene 81

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. One of the major causes of the high attrition rate is the poor predictive value of current preclinical models used in drug development. In response to these challenges, the newly passed FDA Modernization Act 2.0

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

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. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

Development 52

Development Drugs In-Vitro Gene Therapy 52

Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

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In-Vitro In-Vivo Sales Clinical Trials 279

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. While the main thrust of the new alliance is finding in vivo therapies, Bayer said it is also working with Mammoth on ex vivo projects “on a nonexclusive basis.”

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

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.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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).

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Gene Editing Gene Gene Therapy In-Vivo 142

The Pharma Data

JANUARY 17, 2021

Previously Rexgenero Ltd, a UK-based company pioneering the development of cell therapies to treat serious diseases such as cancer and chronic limb-threatening ischaemia (CLTI), the launch of Ixaka follows integration of its nanoparticle gene therapy business in France and a shareholder restructuring. A total of $15.4 A total of $15.4

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

Drug Discovery World

APRIL 12, 2023

The decision by the Biden administration to end the federal requirement for drugs under development to be tested on animals represents a significant step forward for the life sciences sectors. The legislation means that drug developers will now have further options to investigate the safety and efficacy of their products under investigation.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

pharmaphorum

SEPTEMBER 28, 2022

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. 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

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

AUGUST 14, 2023

Over the past few decades, the incredible progress in personalised medicine has led to the development of life-saving cell and gene therapies (CGT). For instance, in a trial where patients are selected based on a specific human leukocyte antigen (HLA) type, the FDA requires the use of a fully certified, FDA approved Companion Diagnostic.

Gene Therapy 52

Gene Therapy Marketing Gene DNA 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. I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. Most common health conditions already have medications to treat them.

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In-Vivo Generic Drugs Gene Therapy Drugs 187

The Pharma Data

MAY 3, 2021

In its 2020 annual report, the division of the US Food and Drug Administration (FDA) tasked with addressing emerging regulatory issues and developing new tools and approaches for regulatory reviews said it was able to expand its efforts despite the COVID-19 pandemic impacting most of the year. . Posted 03 May 2021 | By Michael Mezher .

In-Vitro 52

In-Vitro Clinical Trials Clinical Trials In-Vivo 52

pharmaphorum

OCTOBER 6, 2022

Thirty years on, GWI has proven itself to be a challenging clinical condition; chronic and multisymptomatic by nature, the disease affects around a third of all veterans who served during the Gulf War. A complex range of factors are thought to contribute to the development of the illness. Disease modelling Gulf War Illness.

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In-Vivo Research Genetics Drugs 98

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. Those funds were used to advance its discovery and development programs.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 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. Genetically modified T cells stimulated with IL2 are now administered as cell therapy for multiple cancer cell types with beneficial results 3,4. This continues to be the standard of care.

Research 52

Research In-Vitro In-Vivo Development 52

Pharmaceutical Technology

APRIL 12, 2023

The Association of the British Pharmaceutical Industry (ABPI) also states that “the use of animals in medical research has played and continues to play an essential role in the development of new medicines and vaccines”. This is to ensure patient safety, as outlined in The Medicines for Human Use (Clinical Trials) Regulations 2004.

Regulation 245

Regulation Vaccination Vaccine Medicine 245

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

Drug Discovery World

APRIL 4, 2023

Read more: New oral option for anaemia of CKD approved in US EA-2353, Endogena Therapeutics The investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) was designated as a Fast Track development programme in February. Here’s a summary. The drug was granted orphan drug designation by the FDA in May 2021.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

FEBRUARY 6, 2023

WM : Infinimmune is an early-stage antibody biotechnology company, focused on reinventing antibody drug discovery by developing human-derived antibody drugs. However, the limited number of drugs and targets demonstrates the need for new approaches to both discovery and development. This has consequences for safety and efficacy.

Antibody 52

Antibody Immune Response Genome Genomics 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

Drug Discovery World

JULY 18, 2023

Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19. Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies.

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DNA Genome Genomics RNA 98

The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5

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Gene Therapy Gene In-Vivo Licensing 40

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 ”. For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. to £104.7m

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Roots Analysis

AUGUST 31, 2023

An additional level of genetic control is provided by gene switches that are located upstream of the promoter region. These genetic switches assist transcription factors in binding to the promoter region. This is true for any genetic products or byproducts of the metabolic pathways that are toxic to the host.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

pharmaphorum

OCTOBER 8, 2020

Scribe – which counts new Nobel Prize for chemistry winner Jennifer Doudna among its founders – is getting $15 million upfront from Biogen for an alliance focusing on the development of a CRISPR-based therapy for neurodegenerative disease including amyotrophic lateral sclerosis (ALS). Benjamin Oakes.

Gene Editing 54

Gene Editing Gene In-Vivo Clinical Trials 54

Drug Discovery World

JANUARY 23, 2023

Cargene Biopharma A company developing highly specific, stable, potent, siRNA-based oligonucleotide therapeutics via the company’s OSCAR algorithm and SMoP pharmacophore design platform. Our initial focus is to develop mRNA therapies that act through multiple immune mediated mechanisms to deliver potentially curative treatments in oncology.

RNA 98

RNA Genome Genomics Engineer 98

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.

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Research Protein Scientist Immune Response 98