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. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

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Gene Therapy Gene Gene Editing Clinical Trials 52

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. The move marks a continuation in its ongoing commitment to exploring gene-editing technology. DOWNLOAD THE FULL ARTICLE HERE.

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Gene Editing Gene Gene Therapy Genetics 52

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 97

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. Roughly 80% of rare diseases are due to a known genetic driver.

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Gene Gene Editing Gene Therapy Clinical Trials 130

Pharmaceutical Technology

APRIL 25, 2023

After missing the previous Q1 deadline, bluebird bio has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for its sickle cell disease gene therapy lovo-cel, based on an April 24 company announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Gene Editing Licensing 130

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

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.

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Gene Therapy Gene Production Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced.

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Gene Therapy Gene Gene Editing Clinical Trials 52

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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Research Gene Editing Gene Genetics 118

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. Many novel therapies have been successfully developed through such partnerships.

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Research Gene Editing Development Gene 132

pharmaphorum

DECEMBER 14, 2022

Boyds hires cell and gene therapy expert. Drug development consultancy Boyds has tapped Dr Patrick Ginty as senior director of regulatory affairs. Ginty is an expert in cell and gene therapies and has previously done regulatory affairs work at AviadoBio and UCB-subsidiary Handl Therapeutics. Other agency hires.

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Life Science Gene Therapy Gene Editing Gene 98

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

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

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.

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In-Vivo Gene Editing Trials Gene 52

pharmaphorum

FEBRUARY 17, 2022

Global Blood Therapeutics’ run of positive regulatory news for its oral sickle cell disease treatment Oxbryta has continued with an approval for the drug in Europe for patients aged over 12. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

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.

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Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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Gene Editing Gene In-Vivo Genetics 52

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

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. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

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 In-Vivo 40

Delveinsight

FEBRUARY 25, 2021

For most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often long-lasting and with considerable expense. Gene and cell therapies hold the potential to provide a one-off cure. Besides, the therapy area also suffers from regulatory challenges. Race to be the first mover.

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Gene Gene Therapy Gene Editing Development 52

Delveinsight

SEPTEMBER 14, 2021

ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. Vor Biopharma’s AML drug secures Fast Track Designation. ADARx bags USD 75 Million to advance its RNA tech pipeline. Carl Gordon, Ph.D.,

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RNA Clinical Trials Clinical Trials Drugs 67

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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Gene Editing Manufacturing Gene In-Vivo 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

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Business Development Development Gene Editing In-Vivo 40

pharmaphorum

OCTOBER 18, 2021

Already struggling to make headway with its recently launched Alzheimer’s drug, Biogen suffered a major late-stage pipeline setback after its tofersen drug for amyotrophic lateral sclerosis (ALS) missed the mark in a late-stage study. 1/2) [link]. — MND Association (@mndassoc) October 17, 2021.

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Trials Gene Editing Gene Gene Therapy 45

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. That would just slow the whole field down.

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Gene Editing Gene Gene Therapy In-Vivo 145

The Pharma Data

DECEMBER 28, 2020

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Dr. Russell has managed all aspects of the drug development and regulatory approval processes for oncology and non-oncology drug candidates. from the Harvard T.H.

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Gene Therapy Life Science Gene Gene Editing 52

The Pharma Data

NOVEMBER 3, 2020

Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. About LogicBio Therapeutics.

Clinical Trials 40

Clinical Trials Clinical Trials Gene Editing Genome 40

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. Beqvez is also being reviewed by the US Food and Drug Administration (FDA) with a decision anticipated by mid-2024.

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Gene Therapy Gene Gene Editing Sales 111

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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Research Gene Gene Therapy Genome 264

Pharmaceutical Technology

AUGUST 24, 2022

The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for iECURE’s GTP-506 to treat Ornithine Transcarbamylase (OTC) deficiency in paediatric patients. A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy.

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Gene Editing Gene Therapy Gene Genetics 130

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A.

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Gene Therapy Gene Gene Editing Genetics 52

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. However, she said the company wants to strengthen its position in gene editing.

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Gene Therapy Gene Gene Editing Licensing 52

pharmaphorum

JULY 1, 2021

Synthetic biology holds much promise for the life sciences sector as it continues to look for new ways of treating disease and accelerating drug development, says Debiopharm’s Tanja Dowe. The next decade is where it really starts to get interesting, as we see more and more ‘synbio’-enabled drugs enter clinical trials.

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Gene Editing Engineer Development Gene 88

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

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Gene Editing Gene Gene Therapy Scientist 105