XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency.

Gene Therapy 105

Gene Therapy FDA Approval Gene Clinical Trials 105

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 252

Gene Therapy Gene Genetics Development 252

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.

Gene Therapy 256

Gene Therapy Gene Development Manufacturing 256

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

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. Sanofi is one of the leading patent filers working with viral vectors.

Gene Therapy 246

Gene Therapy Gene Genomics Genome 246

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

Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

Gene Therapy 281

Gene Therapy Gene Genetics Trials 281

Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

Genetics 328

Genetics Medicine Development Gene Therapy 328

Bio Pharma Dive

FEBRUARY 12, 2021

Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.

Gene Therapy 281

Gene Therapy Gene Genetics Medicine 281

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 Development 243

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.

Medicine 189

Medicine Research Gene Therapy Genetics 189

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 189

Genetics Trials Clinical Trials Clinical Trials 189

Pharmaceutical Technology

FEBRUARY 13, 2023

While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genomics 264

Pharmaceutical Technology

JUNE 22, 2023

Ultragenyx Pharmaceutical has opened its new gene therapy manufacturing facility in Bedford, Massachusetts, US. The selection of Bedford for the new 110,000ft² facility was influenced by the proximity to the company’s research and development operations in the Greater Boston area.

Gene Therapy 130

Gene Therapy Gene Manufacturing Production 130

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

Pharma Mirror

OCTOBER 26, 2021

a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, has formed a new research collaboration with Mayo Clinic. Castle Creek Biosciences Inc., Deyle, M.D.,

Gene Therapy 130

Gene Therapy Gene Genetics Clinical Development 130

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. Vertex remains committed to advancing CF research, with plans to expand the impact of their CFTR modulators and explore new genetic mutations.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

Pharmaceutical Technology

FEBRUARY 24, 2023

AbbVie is expanding its strategic collaboration with Capsida Biotherapeutics for the development of targeted genetic medicines to treat eye diseases with high unmet needs. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 130

Gene Therapy Gene Engineer Manufacturing 130

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. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

BioSpace

JULY 12, 2021

A new study has found potential in a novel gene therapy method to help children born with the rare genetic and neurodegenerative disorder, AADC deficiency.

Gene Therapy 94

Gene Therapy Gene Genetics Research 94

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 97

Gene Therapy Gene Manufacturing Clinical Trials 97

pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

Gene Therapy 105

Gene Therapy Gene Gene Sequencing Immune Response 105

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

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. FDA backing.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

Pharmaceutical Technology

MAY 17, 2023

The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. CircRNA research and development activities are being conducted at the company’s facility at the Karolinska Institute in Stockholm, Sweden.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

FEBRUARY 20, 2023

While there is no cure to this neurodegenerative condition, academics and companies are pushing through with research that could help patients and their families. Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials.

Development 246

Development Gene Therapy Protein Gene 246

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

Gene Editing 126

Gene Editing Gene Genetic Disease Research 126

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? Zolgensma Gene Therapy: START Trial.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

STAT News

AUGUST 8, 2022

Scientist Jim Wilson is synonymous with gene therapy — and for good reason. For decades, the celebrated researcher has been a pioneer in the field, focused on the development of treatments for rare, often overlooked diseases.

Gene Therapy 98

Gene Therapy Gene Scientist Research 98

FDA Law Blog

MARCH 13, 2023

To further expand patient input into product development, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products or “OTP” (formerly the Office of Tissues and Advanced Therapies, or “OTAT”) is holding a free public workshop titled, Clinical Trials: The Patient Experience.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.

Development 241

Development RNA Gene Therapy Genetics 241

Worldwide Clinical Trials

NOVEMBER 27, 2023

In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023. Why is Casgevy a Breakthrough Gene Therapy for Patients with Sickle Cell Disease?

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189