Pharmaceutical Technology

APRIL 15, 2024

Discover the groundbreaking gene therapy patent by Ginkgo BioWorks Inc for treating NPC1 using AAV vectors. Learn how this innovative approach offers hope for rare genetic disorders like Neimann-Pick Disease Type C.

Gene Sequencing 130

Gene Sequencing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

FEBRUARY 8, 2024

Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant to invest in cell and gene therapy.

Gene Therapy 246

Gene Therapy Gene Medicine Development 246

Pharmaceutical Technology

DECEMBER 21, 2023

Genevoyager has announced the opening of contract development and manufacturing organisation facility to manufacture gene therapy products.

Gene Therapy 243

Gene Therapy Gene Development Manufacturing 243

Bio Pharma Dive

NOVEMBER 13, 2023

Forge Biologics operates a contract manufacturing business as well as develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.

Gene Therapy 278

Gene Therapy Gene Contract Manufacturing Manufacturing 278

Pharmaceutical Technology

APRIL 2, 2024

In this issue: High-profile gene therapies that use AAV vectors, new research into HIV-TB coinfection treatments, traceability technologies for the supply chain, and more.

Gene Therapy 130

Gene Therapy Gene Research 130

Bio Pharma Dive

OCTOBER 5, 2023

Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.

Gene Therapy 268

Gene Therapy Gene Marketing 268

Pharmaceutical Technology

DECEMBER 7, 2023

The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD.

Gene Therapy 264

Gene Therapy Gene 264

Pharmaceutical Technology

JANUARY 17, 2024

The US FDA has approved Vertex's CASGEVY, a gene-edited cell therapy, to treat transfusion-dependent beta thalassemia.

Gene Editing 245

Gene Editing Gene FDA Approval 245

Pharmaceutical Technology

NOVEMBER 20, 2023

The value of haemophilia gene therapies like Roctavian and Hemgenix hinge on long-term efficacy and innovative payment structures play a key role.

Gene Therapy 246

Gene Therapy Gene 246

Pharmaceutical Technology

NOVEMBER 30, 2023

As more cell and gene therapies get approved to treat common conditions, scaling them up will be a challenge.

Gene Therapy 264

Gene Therapy Gene Manufacturing 264

Pharmaceutical Technology

JANUARY 29, 2024

The full potential of cell and gene therapies (CGTs) has not yet been discovered as these therapies promise life-changing treatments.

Gene Therapy 244

Gene Therapy Gene 244

Bio Pharma Dive

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

Gene Therapy 169

Gene Therapy Gene FDA Approval Development 169

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

Bio Pharma Dive

JANUARY 6, 2024

The Emeryville, California- based gene therapy developer has several preclinical programs in development with Moderna, Ionis and Affini-T.

Gene Editing 208

Gene Editing Gene Gene Therapy Development 208

Pharmaceutical Technology

NOVEMBER 21, 2023

According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapy holds the greatest potential to transform the Gaucher disease landscape

Gene Therapy 241

Gene Therapy Gene Marketing 241

Bio Pharma Dive

APRIL 2, 2023

The startup, a successor to an eye gene therapy biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.

Gene Therapy 328

Gene Therapy Gene 328

Bio Pharma Dive

JUNE 6, 2023

The biotech has raised about $65 million to test two ways to deliver larger genes into the body, each of which could help gene therapy treat more diseases.

Gene 258

Gene Gene Therapy 258

Pharmaceutical Technology

MAY 23, 2023

Avrobio has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m. In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration.

Gene Therapy 258

Gene Therapy Gene Development Drugs 258

Pharmaceutical Technology

OCTOBER 26, 2023

The potential for using gene therapies to treat sickle cell disease is high, but their durability and high price point invite scrutiny.

Gene Therapy 246

Gene Therapy Gene 246

Pharmaceutical Technology

DECEMBER 11, 2023

The FDA has approved Vertex/CRISPR’s Casgevy and bluebird bio’s Lyfgenia gene therapies for the red blood cell disorder.

Gene Therapy 246

Gene Therapy Gene FDA Approval 246

Bio Pharma Dive

MARCH 6, 2023

The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

Gene Therapy 268

Gene Therapy Gene Licensing Licensing 268

Pharmaceutical Technology

OCTOBER 17, 2023

SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.

Gene Therapy 246

Gene Therapy Gene Licensing Licensing 246

Pharmaceutical Technology

AUGUST 16, 2023

Several companies are developing robotics to automate cell and gene therapy manufacturing, but some challenges persist.

Gene Therapy 246

Gene Therapy Manufacturing Gene Development 246

Bio Pharma Dive

OCTOBER 24, 2022

Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.

Gene Therapy 270

Gene Therapy Gene Development 270

Bio Pharma Dive

FEBRUARY 22, 2023

The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”

Gene Editing 257

Gene Editing Gene In-Vivo RNA 257

Bio Pharma Dive

JANUARY 13, 2022

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.

Gene Therapy 352

Gene Therapy Gene Research 352

Pharmaceutical Technology

APRIL 3, 2023

The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.

Gene Therapy 246

Gene Therapy Gene DNA Reagent 246

Pharmaceutical Technology

OCTOBER 19, 2022

Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.

Gene Therapy 298

Gene Therapy Gene Development Genetics 298

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.

Gene Therapy 246

Gene Therapy Gene Trials Clinical Trials 246

Bio Pharma Dive

AUGUST 3, 2023

One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.

Gene Therapy 246

Gene Therapy Gene Sales 246

Bio Pharma Dive

JANUARY 4, 2023

The Lilly-owned subsidiary will pay $55 million to gain access to Capsida’s AAV gene therapy technology in a deal aimed at central nervous system disorders.

Gene Therapy 328

Gene Therapy Gene Development 328

Bio Pharma Dive

APRIL 4, 2023

biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.

Gene Therapy 283

Gene Therapy Gene 283

Pharmaceutical Technology

SEPTEMBER 15, 2023

Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.

Genetics 246

Genetics Gene Gene Therapy Licensing 246

Bio Pharma Dive

JUNE 27, 2023

The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.

Gene Therapy 269

Gene Therapy Gene Marketing 269

Bio Pharma Dive

MARCH 20, 2023

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

Gene Therapy 245

Gene Therapy Gene Development Manufacturing 245

Pharmaceutical Technology

FEBRUARY 10, 2023

4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and large market diseases, has had the FDA place a clinical hold onto its Fabry disease (FD) gene therapy program (4D-310).

Gene Therapy 264

Gene Therapy Gene Trials Marketing 264