Pharmaceutical Technology

APRIL 19, 2024

Paolo Morgese, vice president of European public affairs at the Alliance for Regenerative Medicine, discusses the barriers affecting access to gene therapies.

Gene Therapy 223

Gene Therapy Gene Medicine 223

Pharmaceutical Technology

MARCH 21, 2024

Biotech executives discuss why developing gene therapies for central nervous system disorders remains difficult at a recent conference.

Gene Therapy 246

Gene Therapy Gene Development 246

Pharmaceutical Technology

FEBRUARY 13, 2024

The regulatory greenlight makes it the only gene therapy available in Europe for sickle cell disease and TDT.

Gene Therapy 264

Gene Therapy Gene 264

Pharmaceutical Technology

OCTOBER 11, 2023

Biomarin and CSL Behring’s haemophilia gene therapies have boosted innovation in the field, but assessing their cost effectiveness is less than straightforward.

Gene Therapy 264

Gene Therapy Gene 264

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

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

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

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

pharmaphorum

MAY 2, 2024

The 4th Next Generation Gene Therapy Vectors Summit (June 12-14, Boston), the industry’s only hub for covering the latest advances and innovations in gene therapy vector development.

Gene Therapy 98

Gene Therapy Gene Development 98

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

APRIL 18, 2024

The future of cell and gene therapy access in Europe could be more precarious than across the pond.

Gene Therapy 162

Gene Therapy Gene 162

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

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

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

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

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

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 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 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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 258

Gene Therapy Gene Development Drugs 258

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Bio Pharma Dive

AUGUST 21, 2023

By partnering with an accomplished CTDMO, gene therapy innovators can benefit from guidance and support.

Gene Therapy 246

Gene Therapy Gene Manufacturing 246