Bio Pharma Dive

APRIL 1, 2022

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.

Gene Therapy 275

Gene Therapy Gene Development Genetics 275

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 237

Gene Therapy Gene Development Engineer 237

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 184

Genetics Trials Clinical Trials Clinical Trials 184

Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

Gene Therapy 130

Gene Therapy Genetic Disease Genetics Gene 130

Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

Genetics 130

Genetics Medicine Development In-Vivo 130

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.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

BioPharma Reporter

MAY 13, 2024

Regeneron Pharmaceuticalsâ gene therapy for otoferlin-related hearing loss, a genetic cause of deafness, has restored hearing to normal levels in one deaf child within just 24 weeks.

Gene Therapy 98

Gene Therapy Gene Genetics Development 98

Bio Pharma Dive

MAY 6, 2024

The British drugmaker, which has made genetic medicine a larger priority of late, added $140 million to a deal to develop up to 10 gene and cell therapies.

Genetics 174

Genetics Gene Medicine Development 174

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

Gene Therapy 243

Gene Therapy Gene Gene Editing Manufacturing 243

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. The funding, with participation from Oxford Science Enterprises (OSE), includes AGTC’s acquisition and capital to help accelerate Beacon Therapeutics’ candidate development. The total financing was £96m ($120m).

Gene Therapy 246

Gene Therapy Gene Trials Clinical Trials 246

Medical Xpress

MAY 16, 2023

However, how the brain has developed over the course of evolution and which genes are responsible for the high cognitive abilities is still largely unclear. The better our understanding of the role of genes in brain development, the more likely it will be that we will be able to develop treatments for serious brain diseases.

Development 119

Development Genetics Gene Research 119

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

Gene Editing 184

Gene Editing Gene Gene Therapy In-Vivo 184

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.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease. It prevents epidermis adhesion to the dermis.

Gene Therapy 264

Gene Therapy Gene FDA Approval Manufacturing 264

Bio Pharma Dive

FEBRUARY 25, 2021

Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.

Gene Therapy 246

Gene Therapy Gene Genetics Development 246

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

BioPharma Reporter

OCTOBER 17, 2023

SpliceBio, a genetic medicines company harnessing protein splicing to develop the next generation of gene therapies, has partnered with Spark Therapeutics to develop a gene therapy for an inherited retinal disease.

Gene 59

Gene Gene Therapy Development Genetics 59

Bio Pharma Dive

FEBRUARY 28, 2024

The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.

Gene Editing 166

Gene Editing Gene Genetics Medicine 166

Scienmag

OCTOBER 26, 2020

New genetic sensors, developed by scientists at University of Warwick and Keele University, could function as a lab test device and even as a live monitoring system inside living cells The innovative system can detect when a specific gene in a cell is active – instead of only detecting its presence The technology is based […].

Genetics 75

Genetics Scientist Gene Development 75

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors.

Gene Therapy 246

Gene Therapy Gene Genome Genomics 246

Bio Pharma Dive

JUNE 15, 2021

A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.

Gene Editing 278

Gene Editing Gene Genetics Development 278

Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

Gene Therapy 342

Gene Therapy Gene Genetics Medicine 342

Bio Pharma Dive

AUGUST 24, 2022

The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

Gene Therapy 282

Gene Therapy Bacteria Gene Drugs 282

Pharma Mirror

FEBRUARY 23, 2021

Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility. The post Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site appeared first on Pharma Mirror Magazine. SOMERSET, N.J. SOMERSET, N.J.

Genetics 130

Genetics Manufacturing Gene Therapy DNA 130

Bio Pharma Dive

OCTOBER 24, 2022

The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.

Gene Therapy 258

Gene Therapy Gene Genetics Medicine 258

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. BioMarin may then license the assets fully to develop and commercialise them across the globe.

Development 130

Development Genetic Disease Genetics Gene Therapy 130

Pharmaceutical Technology

FEBRUARY 9, 2023

The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.

Development 241

Development RNA Gene Therapy Genetics 241

Drug Discovery World

JANUARY 19, 2024

UK scientists have uncovered the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. In the future, this knowledge could aid drug development and interventions that reduce the risk of disease. MPNs can also progress into other forms of blood cancer, such as leukaemia.

Genetics 59

Genetics Gene DNA Development 59

Medical Xpress

MARCH 27, 2023

Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases. The article is published in the journal Human Gene Therapy.

Gene Therapy 60

Gene Therapy Gene Development Genetic Disease 60

Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

Genome 130

Genome Genomics Medicine Development 130

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

Gene Therapy 130

Gene Therapy Gene Licensing Licensing 130

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

Genetics 130

Genetics DNA Antibody Genome 130

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science 130

Life Science Genetic Disease Genome Genomics 130

Olympian Clinical Research

SEPTEMBER 27, 2023

While the exact cause of vitiligo remains a subject of ongoing research, genetics has emerged as a significant factor in understanding its onset and progression. In this deep dive, we’ll explore the genetic links associated with vitiligo and address the burning question: Is vitiligo hereditary?

Genetics 52

Genetics Gene Clinical Research Research 52

Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130