Pharmaceutical Technology

MARCH 11, 2024

Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.

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Gene Therapy Gene 299

Bio Pharma Dive

APRIL 26, 2024

The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.

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Gene Therapy Gene Drugs 305

Bio Pharma Dive

JUNE 12, 2024

The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.

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Gene Therapy Gene Trials 293

Bio Pharma Dive

MAY 24, 2024

But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.

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Gene Therapy Gene 301

Pharmaceutical Technology

SEPTEMBER 25, 2024

The funds will advance Genespire’s gene therapy for methylmalonic acidemia, a group of rare metabolic disorders, to Phase I/II trials.

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Gene Therapy Gene Trials 130

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 284

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.

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Gene Therapy Gene Genetics Clinical Research 194

Bio Pharma Dive

AUGUST 1, 2024

Wilson, who founded UPenn’s gene therapy program three decades ago, will step down to start two new spinouts, Gemma Bio and Franklin Biolabs.

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Gene Therapy Gene Research 190

Bio Pharma Dive

SEPTEMBER 19, 2024

Shares dropped by as much as one-quarter Thursday after the biotech shared longer-term results for a gene therapy it’s developing for wet AMD.

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Gene Therapy Gene Development 144

Pharmaceutical Technology

APRIL 19, 2024

The US Food and Drug Administration (FDA) has granted fast-track designation to Bayer subsidiary Asklepios BioPharmaceutical’s (AskBio) investigational gene therapy AB-1002, aimed at treating congestive heart failure (CHF).

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Gene Therapy Gene Drugs 245

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

Bio Pharma Dive

JULY 15, 2024

The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.

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Gene Therapy Gene 190

Pharmaceutical Technology

APRIL 29, 2024

Experts hold scalability challenges and high costs accountable for market failures within the cell and gene therapy landscape.

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Gene Therapy Gene Marketing 246

Pharmaceutical Technology

APRIL 29, 2024

Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ for haemophilia B.

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Gene Therapy Gene FDA Approval Drugs 245

Bio Pharma Dive

JUNE 13, 2024

The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

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Gene Gene Therapy Trials 244

Pharmaceutical Technology

SEPTEMBER 2, 2024

PHC Corporation has introduced a prototype of LiCellGrow, a cell expansion system designed to manufacture cell and gene therapy products.

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Gene Therapy Manufacturing Gene Production 147

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 284

Bio Pharma Dive

MAY 8, 2024

The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.

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Gene Therapy Gene Genetics 201

Pharmaceutical Technology

APRIL 17, 2024

LX2006 consists of adeno-associated virus (AAV) vector carrying the frataxin gene, responsible for causing Friedreich’s ataxia.

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Gene Therapy Gene 246

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

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 273

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

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

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

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

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 332

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 277

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

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 274

Pharmaceutical Technology

JULY 24, 2024

Merck has entered a non-binding MoU with the Gene Therapy Research Institution (GTRI) to produce a gene therapy for Parkinson's disease.

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Gene Therapy Gene Development Research 130

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

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 261

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 353

Bio Pharma Dive

MAY 2, 2024

Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in gene editing research in recent years.

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Gene Editing Gene Research 157

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 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

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.

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Gene Therapy Gene Medicine 237