Pharmaceutical Technology

MARCH 21, 2024

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

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Gene Therapy Gene Development 246

Bio Pharma Dive

JANUARY 18, 2024

Called Advanced Medicine Partners, the new company will offer specialized cell and gene therapy manufacturing services to biotech and pharma clients.

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Gene Therapy Manufacturing Gene Medicine 273

Pharmaceutical Technology

MARCH 15, 2024

Anocca has entered a licensing agreement with EmendoBio for the use of the latter’s OMNI-A4 nuclease, a gene editing technology.

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Gene Editing Licensing Licensing Gene 244

Pharmaceutical Technology

FEBRUARY 13, 2024

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

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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.

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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.

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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.

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Gene Therapy Gene Contract Manufacturing Manufacturing 273

Pharmaceutical Technology

FEBRUARY 8, 2024

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

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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.

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Gene Therapy Gene 246

Pharmaceutical Technology

JANUARY 17, 2024

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

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Gene Editing Gene FDA Approval 245

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.

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Gene Therapy Gene 264

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.

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Gene Therapy Gene Marketing 264

Pharmaceutical Technology

NOVEMBER 30, 2023

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

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Gene Therapy Gene Manufacturing 264

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.

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Gene Therapy Gene Development Manufacturing 256

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.

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Gene Therapy Gene 244

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.

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Gene Therapy Gene Research 130

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

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Gene Therapy Gene Marketing 241

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.

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Gene Editing Gene Gene Therapy Development 202

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.

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Gene Therapy Gene 323

Pharmaceutical Technology

OCTOBER 17, 2023

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

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Gene Therapy Gene Licensing Licensing 246

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.

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Gene Therapy Gene Development Drugs 258

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.

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Gene Therapy Gene FDA Approval Development 164

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.

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Gene Gene Therapy 256

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.

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Gene Therapy Gene 246

Pharmaceutical Technology

APRIL 18, 2024

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

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Gene Therapy Gene 130

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.

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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.

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Gene Therapy Gene Licensing Licensing 263

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.

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Gene Therapy Gene Development 264

BioSpace

APRIL 21, 2024

From gene-corrected cell therapies to a new CAR-T, the cell and gene therapy space looks to expand its reach into the market.

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Gene Therapy Gene Marketing 119

Pharmaceutical Technology

AUGUST 16, 2023

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

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Gene Therapy Manufacturing Gene Development 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.

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Gene Therapy Gene Development Genetics 298

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.”

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Gene Editing Gene In-Vivo RNA 255

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.

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Gene Therapy Gene Research 351

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.

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Gene Therapy Gene DNA Reagent 246

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.

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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.

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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.

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Gene Therapy Gene Sales 246