pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. The post Roctavian okay sets up another gene therapy test for Europe appeared first on. million spread over five years.

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Pharmaceutical Technology

DECEMBER 8, 2022

According to the revised deal, Daiichi Sankyo and Kite will transfer the Marketing Authorization for Yescarta to Gilead’s Japan subsidiary, Gilead Sciences K.K., The therapy’s sales and promotion works in Japan following the transfer of marketing authorisation will be carried out by the Kite Cell Therapy Business Unit at Gilead Sciences K.K.

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pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

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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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BioPharma Reporter

JANUARY 10, 2023

Catalent is to support the manufacture of Sarepta Therapeutics' gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD).

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STAT News

JULY 29, 2022

Sarepta Therapeutics said Friday that it intends seek regulators’ approval for its gene therapy to treat Duchenne muscular dystrophy earlier than expected — a risky move but one that could help the company reach the market much faster. In early Friday trading, Sarepta shares rose 12% to $96.50.

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BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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BioPharma Reporter

AUGUST 24, 2023

Cell and Gene Therapy Catapult, Rentschler Biopharma, and Refeyn have announced a new partnership to develop and apply âinnovativeâ process analytical technologies (PAT) to improve the process and efficiency of AAV manufacture.

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BioPharma Reporter

NOVEMBER 17, 2020

The future of treatment for many of the most challenging diseases in the world is said to reside in personalized cell and gene therapy (CGT). These expensive and complex therapies have proven highly effective in multiple cases, but a challenge remains around producing them at scale and at a reasonable cost.

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BioPharma Reporter

JANUARY 31, 2023

UK based, Aptamer Group, and Hong Kong headquartered, BaseCure Therapeutics, are paring up on the development of Optimer-targeted gene therapies.

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BioPharma Reporter

APRIL 6, 2023

In an effort to improve its cash runway, the UK biotech company Freeline Therapeutics has paused the development of a gene therapy candidate for the rare metabolic condition Fabry disease. Instead, the company will refocus its efforts on a gene therapy for a different metabolic condition called Gaucher disease.

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BioPharma Reporter

SEPTEMBER 5, 2024

The European Commission has granted Orphan Drug Designation for Vivet Therapeuticsâ gene therapy product VTX-806 for the treatment of Cerebrotendinous Xanthomatosis (CTX).

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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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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BioPharma Reporter

MAY 30, 2024

Cellular Origins and the Cell and Gene Therapy Catapult (CGT Catapult) have announced a new collaboration to demonstrate universal automation of CGT manufacturing.

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BioPharma Reporter

FEBRUARY 21, 2023

Three months after receiving the US green light, CSLâs candidate Hemgenix has become the first gene therapy to gain EU approval for the treatment of hemophilia B.

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Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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BioPharma Reporter

SEPTEMBER 13, 2021

AbbVie is aligning with Regenxbio to develop and commercialize a potential one-time gene therapy for the treatment of chronic retinal diseases.

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BioPharma Reporter

DECEMBER 22, 2021

There is a healthy appetite for continued investment in gene therapy, in new modalities and more indications, says an industry insider as he reflected on the prospects for the US gene therapy sector next year.

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BioPharma Reporter

APRIL 6, 2022

The FDA has released two draft guidance documents: for human gene therapy products incorporating human genome editing, and for CAR T cell products.

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BioPharma Reporter

APRIL 29, 2021

Adverum Biotechnologies has reported a setback in its trial exploring gene therapy for the treatment of diabetic macular edema (DME).

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BioPharma Reporter

SEPTEMBER 23, 2021

This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindnessâ venture arm, the Retinal Degeneration Fund (RD Fund).

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BioPharma Reporter

MARCH 2, 2023

Spark Therapeutics, a Roche company, has officially started work on its US$575m gene therapy innovation center in West Philadelphia. The center is expected to be completed by 2026.

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BioPharma Reporter

FEBRUARY 27, 2023

Chinese companies Porton Advanced Solutions and Yinjia Biosciences have signed a pact aimed at supporting further development of cell and gene therapies.

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BioPharma Reporter

JULY 26, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, is teaming up with Roche to develop novel AAV gene therapy vectors for eye diseases.

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Gene Therapy Gene Development Regulation 98

BioPharma Reporter

DECEMBER 22, 2021

Novartis will acquire UK-based Gyroscope Therapeutics, the company behind a one-time investigational gene therapy for geographic atrophy (GA).

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BioPharma Reporter

MARCH 6, 2023

Sarepta Therapeutics would seem to be edging closer to accelerated FDA approval of its gene therapy for Duchenne muscular dystrophy (DMD).

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BioPharma Reporter

MARCH 25, 2022

Exothera, a CDMO specializing in the industrialization of vaccine and gene therapy processes, reports that it has achieved GMP certification for its facilities in Jumet, Belgium.

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BioPharma Reporter

JANUARY 4, 2022

ATMPS Ltd, a UK developer of blockchain-based cell orchestration platforms for advanced therapies is collaborating with US quality and compliance consultancy, Assurea LLC, to develop a fully complaint blockchain approach for end-to-end supply chain management of cell and gene therapies.

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BioPharma Reporter

JUNE 21, 2022

The developer says it is harnessing parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity.

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BioPharma Reporter

APRIL 24, 2023

bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).

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BioPharma Reporter

SEPTEMBER 16, 2020

CPhIâs Annual Report 2020 predicts impending cell and gene capacity shortages for the US market, and possible mAb production growth in China.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. 1] BioNews. Hemophilia News Today. link] [2] Tang Q, et al.

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BioPharma Reporter

SEPTEMBER 3, 2020

AskBio, which develops adeno-associated virus (AAV) gene therapies for genetic disorders, has won an R&D grant valued at Â2m (around US$2.7m) from Scottish Enterprise.

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BioPharma Reporter

JUNE 29, 2023

Astraveus, the creator of modular, microfluidic cell foundries for cell and gene therapy (CGT) manufacturing, has raised â16.5 million in series seed financing.

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pharmaphorum

NOVEMBER 20, 2020

The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on. Access the official agenda for more information.

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pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

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