pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

Gene Therapy 135

Gene Therapy Gene Marketing Genetic Disease 135

Pharma Mirror

MAY 7, 2021

The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinical trial services to meet the increasing global demand predicted to reach $17.4 The post Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4

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Clinical Trials Clinical Trials Gene Marketing 130

Bio Pharma Dive

MARCH 20, 2024

million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25

Gene Therapy 205

Gene Therapy Gene Genetics Medicine 205

Worldwide Clinical Trials

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. The global nature of cell and gene therapy trials adds another layer of complexity.

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Gene Therapy Clinical Supply Trials Gene 182

Pharmaceutical Technology

MAY 3, 2023

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM.

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Gene Therapy Gene Protein Trials 242

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

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Gene Therapy Gene Genotype In-Vivo 246

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP. By Cytiva Thematic.

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Gene Editing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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RNA Gene Expression Gene Gene Therapy 245

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 114

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Pharma Mirror

JANUARY 30, 2022

Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C&GT) from taking its place as an established treatment area. The global market is projected to reach $13.8 He explains how we can navigate obstacles to ensure C&GT can achieve its full potential.

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Gene Therapy Gene Clinical Development Marketing 130

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.

Gene Therapy 105

Gene Therapy Gene FDA Approval Genetics 105

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.

Gene Therapy 99

Gene Therapy FDA Approval Gene Genetics 99

XTalks

JULY 19, 2021

Phosphorus, a leading preventative genomics company, has developed the first comprehensive preventative genetic test for consumers. The test is called GeneCompass and features medical-grade technology to provide a holistic assessment of genetic health and wellness. The Phosphorus GeneCompass test has a list price of $249.

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Genetics Genome Genomics Gene 98

Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. For cell and gene therapy applications, you need a variety of speciality enzymes of the highest purity, specificity, and consistency. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior.

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Drugs Gene Therapy Production Manufacturing 295

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

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Genetics Drugs Genome Project Biobanking 98

pharmaphorum

MAY 23, 2022

PTC Therapeutic’s Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for gene therapies in the bloc. The post PTC gets green light for gene therapy Upstaza in EU appeared first on.

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Gene Therapy Gene Marketing Genetics 101

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Gene Editing Gene DNA Gene Therapy 235

Pharmaceutical Technology

FEBRUARY 24, 2023

AbbVie is expanding its strategic collaboration with Capsida Biotherapeutics for the development of targeted genetic medicines to treat eye diseases with high unmet needs. In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner.”

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Gene Therapy Gene Engineer Manufacturing 130

STAT News

APRIL 3, 2023

The first CRISPR treatment for sickle cell disease has been submitted to the Food and Drug Administration, beating a rival gene therapy that’s been delayed due to a manufacturing issue.

Genetics 131

Genetics Gene Therapy Gene Manufacturing 131

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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Gene Therapy Gene Genetics Regulation 95

Pharmaceutical Technology

JUNE 29, 2022

The European Commission (EC) has granted marketing authorisation for Gilead Sciences’ subsidiary Kite’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel), to treat adults with relapsed or refractory follicular lymphoma (FL). . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Clinical Trials Clinical Trials Genetics 278

Pharmaceutical Technology

JANUARY 16, 2023

It comprises autologous T cells that are modified genetically with a CAR including a complete human anti-BCMA single-chain fragment variant that has a high binding affinity. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Medicine Gene Therapy Production Gene 298

Pharmaceutical Technology

FEBRUARY 9, 2023

Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications. Asieris Pharmaceuticals discovers, develops, and markets advanced therapeutics to treat GU tumours and other related diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development RNA Gene Therapy Genetics 241

Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Pharmaceutical Technology

DECEMBER 12, 2022

It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma. The development, clinical trial and marketing expenses for the cell therapy will be shared by the companies while co-commercialising the product and equally splitting profits from the US region.

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Development Gene Therapy Clinical Trials Clinical Trials 264

pharmaphorum

MAY 18, 2022

SwanBio Therapeutics has raised $56 million in a second-round financing that will be used to advance its lead gene therapy candidate for adrenomyeloneuropathy (AMN) into clinical testing later this year. The therapy is designed to compensate for the disease-causing ABCD1 gene.

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Gene Therapy Gene Trials Clinical Trials 98

Pharmaceutical Technology

NOVEMBER 7, 2022

Based in the US, NKGen develops and markets new autologous, allogeneic and CAR-NK NK cell therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity.

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Gene Therapy Clinical Trials Clinical Trials Trials 246

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

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Gene Therapy Gene Genetics Trials 105

World of DTC Marketing

JUNE 5, 2021

Obesity is a multifactorial disease in which environmental conditions and several genes play an important role in developing this disease. Obesity and genetics. Over a hundred genes and gene variants related to excess weight have been discovered. The rate of new cases of colorectal cancer dropped 23% during this time.

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Medicine Gene Genetics Research 193

pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. After two years’ follow-up, 90% of the boys given the gene therapy showed minimal loss of function and were still alive.

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Gene Therapy Gene FDA Approval Clinical Trials 95

pharmaphorum

SEPTEMBER 30, 2022

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the middle of 2023. It is administered as a single intravenous dose and uses an adeno-associated virus (AAV) vector to deliver the gene into muscle cells. .”

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Gene Therapy Gene Genetics Protein 105

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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Gene Therapy Gene Genetics Development 74

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Gene Therapy Gene FDA Approval Antibody 98

BioPharma Reporter

JANUARY 2, 2023

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.

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Gene Therapy Genetics Gene Production 118