BioPharma Reporter

JUNE 23, 2022

BIO, the worldâs largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.

Gene Therapy 59

Gene Therapy Genome Genomics Gene 59

Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

Gene Therapy 59

Gene Therapy Gene Bioinformatics Life Science 59

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

Gene Therapy 64

Gene Therapy Gene In-Vitro Development 64

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.

Genome 92

Genome Genomics Medicine Development 92

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

Gene Therapy 105

Gene Therapy Gene Genome Genomics 105

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy 84

Gene Therapy Gene Gene Editing DNA 84

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

SEPTEMBER 9, 2022

US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developing RNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored.

RNA 246

RNA Development Gene Therapy Gene 246

Drug Discovery World

JULY 3, 2023

Cytiva and BioCentriq, a New Jersey-based cell and gene therapy contract development and manufacturing organisation (CDMO), have been awarded funding from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), a public-private partnership whose mission is to accelerate biopharmaceutical innovation.

Gene Therapy 52

Gene Therapy Gene Marketing Manufacturing 52

Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Gene Therapy 52

Gene Therapy Gene Life Science Bioinformatics 52

Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

Gene Therapy 67

Gene Therapy Gene Genome Genomics 67

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

Gene Therapy 109

Gene Therapy Gene Trials FDA Approval 109

Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

Gene Therapy 52

Gene Therapy Gene Manufacturing Life Science 52

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. .

Gene Therapy 52

Gene Therapy Gene Genetic Disease Genetics 52

Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Scribe is also entitled to receive potential payments worth over $1bn on meeting development and commercial milestones.

Development 130

Development In-Vivo Genome Genomics 130

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

Pharmaceutical Technology

OCTOBER 11, 2022

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. Ochre Bio will also expand its platforms for drug discovery and development that include a new RNA chemistry and an exploratory platform for expanding its research to more liver diseases.

RNA 130

RNA Development Genome Genomics 130

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.

Genome 40

Genome Genomics Gene Gene Editing 40

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.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy Gene Genetics Genetic Disease 52

pharmaphorum

APRIL 19, 2021

French genomic medicines firm SparingVision has agreed to buy GAMUT Therapeutics, a biotech specialising in gene therapies for inherited eye diseases such as retinitis pigmentosa (RP) that could compete against Roche/Spark’s Luxturna in a wider patient group. . million) fundraiser in October to develop SPVN06.

Gene Therapy 104

Gene Therapy Gene Production Genome 104

BioPharma Reporter

MAY 7, 2024

Broken String Biosciences, a genomics company driving cell and gene therapy developing, has entered a research collaboration with the Francis Crick Institute, a biomedical discovery institute dedicated to understanding the biology underlying health and disease.

Gene Therapy 52

Gene Therapy Research Genome Genomics 52

The Pharma Data

SEPTEMBER 21, 2020

. A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer. The funding will be used to develop a pipeline of ‘highly selective’ gene therapies targeting cancer stem cells in a variety of solid tumour types.

Gene Therapy 52

Gene Therapy Gene Development Genome 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.

Life Science 162

Life Science Development Gene Therapy Genetics 162

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 52

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.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

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.

Genome 101

Genome Genomics Clinical Trials Clinical Trials 101

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. But now, the increased efficiency is likely making drug development cheaper.

Gene Therapy 52

Gene Therapy Gene Manufacturing Development 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy 52

Gene Therapy Gene Trials Gene Expression 52

Drug Discovery World

JANUARY 26, 2023

DDW Editor Reece Armstrong takes a look at the pharmaceutical research and development market in 2023 and asks key opinion leaders what challenges and trends will emerge throughout the year. billion showed the pharma giant taking further steps into the oncology market and developing its speciality medicines vaccines portfolio.

Development 98

Development Drugs Clinical Trials Clinical Trials 98

Worldwide Clinical Trials

JANUARY 24, 2024

Why Partnering with Genetic Counselors May Benefit Your Clinical Program The American College of Medical Genetics (ACMG) has issued a statement that geneticists should be involved in the development of genetic therapies. As of July 2023, the American Society of Cell + Gene Therapy stated there were 3,905 therapies in development.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

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. It is the year 2030. Alternatively, there are vaccines ready to enter decentralised third-phase clinical trials, so you can enrol quickly.

Genome 342

Genome Genomics Clinical Trials Clinical Trials 342

Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. The company is also eligible for development and commercial milestone payments exceeding $1.5bn.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130