Bio Pharma Dive

SEPTEMBER 23, 2024

Regulatory and strategic challenges can delay the delivery of life-changing cell and gene therapies.

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Gene Therapy Gene 246

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 298

Pharmaceutical Technology

NOVEMBER 14, 2024

The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.

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Gene Therapy Gene Drugs 130

Bio Pharma Dive

NOVEMBER 14, 2024

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review voucher for PTC.

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Gene Therapy Gene 141

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 286

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 294

Pharmaceutical Technology

OCTOBER 25, 2024

Dyno has formed a new research partnership with Roche on the development of AAV gene therapy vectors for neurological diseases.

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Gene Therapy Gene Development Research 147

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 278

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

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

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

NOVEMBER 8, 2022

Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.

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Gene Therapy Gene Development Genetics 305

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 183

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

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 278

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

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

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

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

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 268

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 195

Pharmaceutical Technology

OCTOBER 23, 2024

Ocuphire Pharma has announced the acquisition of Opus Genetics to develop gene therapies for inherited retinal diseases (IRDs).

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Gene Therapy Genetics Gene Development 130

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 328

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 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 270

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 183

Fierce Pharma

NOVEMBER 14, 2024

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.

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Gene Therapy Gene FDA Approval Branding 118

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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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 352

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

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

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Gene Therapy FDA Approval Gene Genetics 115

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 268

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.

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Gene Therapy Gene Development Drugs 258

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

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

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