Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA. The system comprises the Cas9 enzyme and a guide RNA.

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XTalks

FEBRUARY 4, 2025

Related: FDA Approves New Use for Ozempic to Reduce the Risks of Kidney Disease Maze stands out in the biotech space with its Compass platform, which analyzes genetic data to identify disease-linked variants. For complex diseases like chronic kidney disease (CKD), where genetics play a significant role, such a platform holds promise.

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Clinical Trials Clinical Trials Trials Genetics 66

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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Development Genetic Disease Genetics Gene Therapy 130

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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Gene Therapy Gene Trials FDA Approval 111

Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.

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Development RNA Gene Therapy Genetics 241

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B. Additional key data is to be presented at scientific conference early this year.

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Gene Therapy Gene Medicine Trials 98

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Gene Therapy Gene FDA Approval Production 119

STAT News

MARCH 9, 2023

A decade ago, I had pegged gene and cell therapies as the next frontier. In the spring of 2013, I had just visited a lab focused on gene and cell therapies at Baylor College of Medicine. As an investment banker, I was sure they would someday change how patients were treated. Read the rest…

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Gene Gene Therapy Licensing Licensing 98

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

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Gene Therapy Gene Protein Clinical Trials 147

Pharmaceutical Technology

AUGUST 24, 2022

Lacerta Therapeutics has entered a new licensing and research partnership agreement with Eli Lilly and Company subsidiary, Prevail Therapeutics, to discover and develop adeno-associated virus (AAV) capsids for treating central nervous system (CNS) diseases. .

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Development Gene Therapy Genetics Gene 130

Pharmaceutical Technology

APRIL 6, 2023

Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. The new capabilities and IP will be incorporated under Ginkgo’s end-to-end AAV gene therapy development platform.

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Engineer Gene Therapy Gene Manufacturing 130

pharmaphorum

OCTOBER 26, 2020

Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio. The company’s most advanced programmes are in Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – Parkinson’s disease and congestive heart failure.

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Gene Therapy Gene Genetic Disease Genetics 59

pharmaphorum

OCTOBER 18, 2022

Eli Lilly has agreed to buy hearing loss gene therapy developer Akouos in a deal that could value the company at around $610 million – if its lead candidate progresses as hoped in the clinic. The buyout – for $12.50 The buyout – for $12.50 There are no approved drug treatments for this type of hearing loss.

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Gene Therapy Gene Genetics FDA Approval 52

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

pharmaphorum

JULY 5, 2022

It can be caused by exposure to loud noises – including prolonged use of earbuds at high volumes – as well as cancer treatment, illnesses, genetic mutations, or aging. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide. An estimated 1.57

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In-Vivo Bioinformatics Genetics Licensing 110

pharmaphorum

MARCH 24, 2022

GSK has participated in an impressive $175 million Seres C financing for LifeMine led by Fidelity Management & Research Co, paying the company a $70 million license fee along with an undisclosed equity investment as part of a “strategic” partnership focusing initially on three targets.

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Medicine Genome Genomics Engineer 105

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin. Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin.

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Gene Therapy Gene FDA Approval DNA 98

pharmaphorum

AUGUST 19, 2021

Novartis’ Rydapt has become the first and only licensed treatment for rare and life-threatening blood disorder systemic mastocytosis (SM) to be cleared for routine NHS use, after getting a green light from NICE. It is caused by a genetic mutation in the KIT gene, which triggers the abnormal proliferation of mast cells.

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Clinical Trials Clinical Trials Licensing Licensing 124

Delveinsight

DECEMBER 17, 2020

Atsena raises USD 55 Million financings for blindness gene therapy. University of Florida (UF) spinout Atsena Therapeutics has completed a USD 55 million financing that will benefit its gene therapy for a common cause of blindness in children into pivotal trials. It is a gene, which codes for a kinase enzyme of the same name.

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Gene Therapy Gene DNA Genetics 96

XTalks

MARCH 21, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall Rett syndrome is typically caused by a genetic mutation on the MECP2 gene. Significant development regression associated with the disease occurs after a period of normal development from six to 18 months of age.

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FDA Approval Gene Therapy Gene Genetics 94

XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

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Branding Gene Editing DNA Production 98

The Pharma Data

JANUARY 3, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta -galactosidase (?

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 21, 2021

The world-leading team of academic researchers will use their newly designed technical approach to assess which proteins are selectively produced by the kalirin gene in the human brain, and how this differs from other human tissues. Dr. The significant gap in treatment options leaves many with poor long-term quality of life and disability.

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Medicine Protein Drugs Research 111

pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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Gene Editing Gene Genetic Engineering DNA 52

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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In-Vivo Gene Editing Gene Therapy Gene 52

The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient’s cells. PHILADELPHIA, Jan.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Pharmaceutical Technology

MARCH 2, 2023

The research using ImmunoGen’s technology will enable the discovery of the agents for use with gene editing. After completion of the research period for each target, Vertex can obtain a global and exclusive license for their research, development, and commercialisation employing the technology for that target.

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Gene Editing Gene Development Research 130

pharmaphorum

JUNE 7, 2022

million licensing agreement with Alectos Therapeutic, as it continues to rebuild a pipeline behind its side-lined Alzheimer’s therapy Aduhelm. It’s not the first time that Biogen has licensed rights to a drug targeting lysosomal function in Parkinson’s disease.

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Licensing Licensing Gene Therapy Genetics 80

The Pharma Data

NOVEMBER 4, 2020

Ltd (“Juyou”), a biotechnology company that develops and sells medical and cosmetic skincare products, for the commercialization and development of Pliaglis ® in mainland China (the “License Agreement”). million and US$1.8 For additional information, please visit [link].

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Licensing Licensing Dermatology Production 40

pharmaphorum

DECEMBER 9, 2022

This means that enrolment is now completed for the initial target or morphological cohort to form the basis of a Biological License Application (BLA) submission by the end of CY2023 to the US Food and Drug Administration (FDA). Syncona has agreed to invest circa $28 million (£22.9 The tender offer for that acquisition was approximately $23.5

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Trials Clinical Trials Clinical Trials Gene Therapy 98

XTalks

SEPTEMBER 17, 2020

Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. Prevalence: 1 in 3,500.

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Trials Gene Hormones Clinical Trials 98

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Gene Therapy Gene Gene Editing Medicine 42

The Pharma Data

DECEMBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Burning Rock Biotech Limited (NASDAQ: BNR, the “Company” or “Burning Rock”) today announced that it entered into an exclusive licensing agreement with Oncocyte Corporation (NYSE American: OCX) to bring DetermaRx , a risk stratification test for early stage lung cancer patients, to China.

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Licensing Licensing Marketing Gene Expression 52

pharmaphorum

AUGUST 7, 2020

Mutations in the LRRK2 gene can cause Parkinson’s disease. Inhibition of LRRK2 activity may slow the progression of Parkinson’s disease in patients with and without known genetic risks based on restoration of lysosomal function.

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Bacteria Marketing Licensing Licensing 69

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

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Gene Silencing Drugs Medicine RNA 59

pharmaphorum

MARCH 25, 2021

Knowing which mutations a tumour needs to survive, and which drugs can target those mutations, is the key to precision oncology, which matches treatment to the genetic profile of a patient’s cancer.

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Drugs Genome Project Genome Genomics 59

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7

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FDA Approval Gene Silencing Drugs RNA 52