Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. In my capstone, I focused on how, if, and when genetic counselors discuss clinical trials with their patients in the context of a counseling session.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Drug Discovery World

MARCH 13, 2023

GenSight Biologics has revealed pooled safety data from five clinical studies of lenadogene nolparvovec (Lumevoq), the largest cohort of ND4-LHON (Leber hereditary optic neuropathy) patients studied after gene therapy treatment.

Gene Therapy 52

Gene Therapy Gene Immune Response Genome 52

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% 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

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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Gene Editing Gene Gene Therapy In-Vivo 142

Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach.

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Gene Therapy Gene Manufacturing Development 52

Pharmaceutical Technology

SEPTEMBER 22, 2022

You have just received the results from your whole genome sequencing test, offered through your public health provider, and discovered that you have a 75% chance of developing a rare form of cancer. Alternatively, there are vaccines ready to enter decentralised third-phase clinical trials, so you can enrol quickly.

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Genome Genomics Clinical Trials Clinical Trials 342

pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. Tailored oncology. In other words, every person’s cancer has its own genetic construct.

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Genome Genomics Clinical Trials Clinical Trials 101

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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Gene Editing Genome Genomics Gene Therapy 101

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

STAT News

JANUARY 30, 2023

The human genome was sequenced in 2003. It is now possible to treat diseases with gene therapy, antisense oligonucleotides, messenger RNA (mRNA), noncoding RNA (known as small interfering RNA, or siRNA), and other gene-based modalities. New ways of conducting clinical trials have also emerged.

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RNA Gene Therapy Drugs Gene 111

pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

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Genome Genomics Medicine Development 92

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

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Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We are excited to lead this financing for Eligo and support the company as they move to the clinic,” said Laia Crespo, Partner at Sanofi Ventures. “We

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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RNA Gene Expression Gene Gene Therapy 245

Drug Discovery World

FEBRUARY 12, 2024

We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials. Matt Todd, Head of Architecture, Ori Biotech “We expect AI and data analytics to support pivotal advances in cell and gene therapy (CGT) manufacturing.

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Genome Genomics Life Science Gene Therapy 139

Drug Discovery World

JANUARY 26, 2023

The modernisation of R&D models could be key to reducing spend on new drugs, a point that Deloitte makes about the need to transform the clinical trials process. At the height of the pandemic, DCTs were seen as a viable alternative to keeping the engines running on clinical trials when non-critical in-person care was paused.

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Development Drugs Clinical Trials Clinical Trials 98

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

DECEMBER 4, 2023

In a statement outlining its investigation, the FDA said it received reports of the T-cell malignancies from clinical trials and post-marketing safety monitoring. The vectors can potentially cause cancer by inserting genetic material into a patient’s genome, particularly near cancer-related DNA sequences or suppressors​​​​.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

XTalks

FEBRUARY 14, 2022

The drug’s approval in the EU was based on analysis of clinical trial data including both pre- and early-symptomatic, and early-onset MLD patients, which showed that a single-dose treatment is effective in modulating disease progression in most early-onset MLD patients. The incidence of MLD is approximately one in 100,000 worldwide.

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Gene Therapy Drugs Gene Gene Silencing 98

XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. Additionally, Gag-specific CD8+ T cells demonstrated an increase of 1.7

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

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Development Gene Therapy RNA DNA 52

Drug Discovery World

MARCH 9, 2023

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

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In-Vivo Gene Editing Gene Trials 52

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

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Life Science Development Gene Therapy Genetics 162

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). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 81

Drug Discovery World

JULY 10, 2023

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. There are myriad ways that this is being enabled. Disappointing, but work is continuing.

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Gene Therapy Gene Scientist Gene Editing 52

Drug Discovery World

MAY 6, 2024

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. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

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Drugs Life Science Gene Editing Gene 59

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. The clinical study also highlighted the favorable tolerability profile of Filsuvez. The most commonly reported adverse reactions in the clinical trial were pruritus (itching) and pain at the wound application site, occurring in 7.3

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

XTalks

MAY 23, 2024

XTALKS CLINICAL EDGE: Issue 2 — Lumos Pharma’s Interview Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. One of the foremost challenges is identifying patients.

Clinical Trials 52

Clinical Trials Clinical Trials Hormones Trials 52

Pharmaceutical Technology

JANUARY 20, 2023

On January 4, the company announced plans to acquire Japanese biotech OriCiro Genomics in an $85 million deal. Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva. More recently, Moderna has begun expanding its scope through global partnerships and deals. said Bancel.

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Manufacturing Vaccination Vaccine Gene Therapy 246

Pharmaceutical Technology

FEBRUARY 20, 2023

If clinical trial participants are representative of the wider disease population, they can help sponsors assess the effectiveness of new and existing therapeutics. of the clinical trial population globally. participation of white subjects. The technology platforms are almost the same.

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Research Clinical Trials Clinical Trials Trials 130

Pharmaceutical Technology

MARCH 29, 2023

Pharmaron is receiving a £151 million investment in capital to support the growth of operations in Liverpool, quadrupling production capacity for gene therapy and vaccine components. On March 10, the National Health Service provided a grant for building a new cell and gene therapy manufacturing facility in Bristol.

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Life Science Manufacturing Gene Therapy Medicine 276

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

NOVEMBER 27, 2023

The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “As

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Protein Gene Medicine Gene Therapy 52