Pharmaceutical Technology

OCTOBER 13, 2022

Vita Therapeutics has raised $31m in a Series B financing round for the development of cell therapies for neuromuscular diseases and cancers. Vita Therapeutics intends to use the funding to advance its lead pre-clinical programme VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to clinical trials.

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Development Gene Editing Gene Gene Therapy 130

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

Gene Editing 52

Gene Editing Development Drugs Clinical Trials 52

Worldwide Clinical Trials

OCTOBER 24, 2023

While this is a small number of people in each rare disease patient community, the significant and continued growth of rare disease drug development is unsurprising given that there are over 10,000 rare diseases , with more being discovered every year.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

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Gene Editing Gene Life Science Regulation 105

BioSpace

OCTOBER 14, 2021

Read about TFF Pharmaceuticals and Augmenta Bioworks' dry powder COVID-19 antibody formula, the world's first gene editing clinical trial for PKU, Bayer's COVID-19 vaccine and other key developments in life sciences research.

Gene Editing 98

Gene Editing Life Science Clinical Trials Clinical Trials 98

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 97

Pharmaceutical Technology

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

Gene Editing 100

Gene Editing Genetic Engineering Engineer Clinical Trials 100

Drug Discovery World

DECEMBER 8, 2023

ONK Therapeutics and NAYA Biosciences have agreed to develop a combination therapy of ONK’s optimally-engineered natural killer (NK) cell therapies and NAYA’s FLEX-NK bispecific antibodies. ONK and NAYA plan to assess several combination therapies in preclinical cancer models in 2024 prior to subsequently exploring initiating clinical trials.

Antibody 52

Antibody Gene Editing Clinical Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Figures from the Amercian Cancer Society 1 state that “a little over 1.9

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Development Research Drugs Antibody 65

Pharmaceutical Technology

MARCH 2, 2023

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Gene Editing 100

Gene Editing Clinical Trials Clinical Trials Genetics 100

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. NTLA-2001 is the furthest along in testing for Intellia.

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In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

OCTOBER 25, 2022

Scientists have shown how stomach cancers can develop resistance to a new class of drugs called ATR inhibitors by switching off the activity of two key genes – raising the possibility of outsmarting cancer by predicting drug resistance in advance. . CRISPR used to identify genes involved. It was funded by Cancer Research UK.

Development 52

Development Drugs Gene Editing Gene 52

Drug Discovery World

MARCH 10, 2023

There have been several other announcements related to clinical trials in the last few days, including positive data from trials in severe influenza and biliary tract cancer, the launch of a study into an ADC treatment for solid tumours, and a new research partnership with the mission to improve clinical trial design.

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In-Vivo Clinical Trials Clinical Trials Drugs 52

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Exposure-Response Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Merck overview Merck, a subsidiary of E.

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Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Head And Neck Cancer Squamous Cell Carcinoma. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

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Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

APRIL 28, 2023

Editas’ CRISPR therapies represent a different approach to the gene editing technology than the one used by CRISPR Therapeutics , which relies on the CRISPR/Cas9 platform. This increases the expression of fetal haemoglobin.

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Gene Therapy Drugs Gene Gene Editing 264

Drug Discovery World

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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Genome Genomics Gene Gene Editing 40

Drug Discovery World

MAY 6, 2024

In this article, DDW’s Diana Spencer highlights several prominent hotspots that are taking the lead on advancing European drug discovery and development. billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1.

Drugs 59

Drugs Life Science Gene Editing Gene 59

Drug Discovery World

NOVEMBER 14, 2023

“I am very encouraged by the initial data from the heart-1 trial that demonstrated the potential for single-course gene editing as a new approach to treat patients with HeFH. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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In-Vivo Gene Editing Gene Genome 52

XTalks

JANUARY 26, 2024

Key breakthroughs at the forefront include novel approaches to cancer treatment and diagnosis, the integration of artificial intelligence (AI) in oncology and the development of advanced preventive measures and care. Overcoming drug resistance: A major challenge in cancer treatment is the development of resistance to chemotherapy drugs.

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Research Gene Editing Gene Genetics 118

Drug Discovery World

DECEMBER 20, 2022

There will be greater emphasis on diversity in clinical trials. A focus on the representation of diverse patient populations in clinical trials will be a very good thing for the industry, but could be challenging. DDW’s Diana Spencer summarises the key trends likely to impact the market in 2023. .

Drugs 52

Drugs Gene Editing Clinical Trials Clinical Trials 52

pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs.

Gene Therapy 52

Gene Therapy Gene Gene Editing Clinical Trials 52

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Gene Therapy Gene Gene Editing Medicine 42

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. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

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In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients – and how to do this faster and more efficiently.

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Research Gene Editing Gene Development 132

XTalks

MARCH 28, 2024

Colorectal Cancer: Advances in Treatment Options In recent years, there has been considerable progress in understanding the biology of CRC, which has helped develop more effective and personalized treatment options. An overview of some of the most promising developments in CRC treatment is provided below.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

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

OCTOBER 24, 2023

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.

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Gene Editing Engineer Clinical Trials Clinical Trials 52

Drug Discovery World

SEPTEMBER 6, 2022

As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . ViaCyte, Beam, and Verve are all due to begin gene editing trials this year. The future of cell and gene therapy: opportunities for innovation .

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Gene Therapy Gene Gene Editing Manufacturing 52

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

XTalks

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

DECEMBER 21, 2023

DDW’s top 10 most popular articles for 2023 reflect the key subjects in drug discovery and development. In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic.

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Gene Therapy Gene Antibody FDA Approval 59

pharmaphorum

JULY 13, 2021

Novo Nordisk also gets rights to the remainder of Dublin-based Prothena’s ATTR amyloidosis programme under the terms of the deal, although PRX004 is the only candidate that has reached the clinical trials stage. All told, the deal could be worth up to $1.2

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Gene Silencing Sales Gene Editing Clinical Trials 98