Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Gene Therapy Gene Development Engineer 237

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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Gene Therapy Gene Licensing Licensing 130

Bio Pharma Dive

DECEMBER 14, 2021

Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress. But gene therapies and other new medicines might provide competition.

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Gene Therapy Licensing Licensing Gene 314

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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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

APRIL 13, 2023

ImaginAb will be responsible for licensing and supplying the clinical doses of 89Zr crefmirlimab berdoxam, its investigational CD8 ImmunoPET tracer, to Leucid Bio. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Licensing Licensing Gene Therapy Gene 130

Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

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Gene Therapy Gene Gene Editing Clinical Trials 111

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 109

FDA Law Blog

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Gene Therapy Gene Trials Clinical Trials 98

Pharmaceutical Technology

JUNE 13, 2023

The US Food and Drug Administration (FDA) approved Ferring Pharmaceuticals’ Prior-Approval Supplement (PAS) to the Biologics License Application (BLA) for the bladder cancer gene-therapy Adstiladrin (nadofaragene firadenovec). The therapy received approval based on positive Phase III trial (NCT02773849) results.

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Gene Therapy Manufacturing Gene FDA Approval 246

BioSpace

MARCH 11, 2021

The European Medicines Agency has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe.

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Gene Therapy Gene Licensing Licensing 91

pharmaphorum

DECEMBER 9, 2021

Dutch biotech uniQure and partner CSL Behring are planning to file for approval of their gene therapy for haemophilia B, armed with new trial data that they say answers earlier concerns about the programme. The therapy combines a viral vector with a gene coding for Factor IX (FIX) which is deficient in patients with haemophilia B.

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Gene Therapy Gene Trials Licensing 52

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.

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Gene Therapy Gene Medicine Trials 96

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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Gene Therapy Gene Genetic Disease Genetics 77

pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. The one-shot therapy aims to treat GA by delivering the gene for complement factor I (CFI) to the eye using an adeno-associated virus (AAV) vector.

Gene Therapy 95

Gene Therapy Gene Licensing Licensing 95

pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

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Gene Therapy Gene DNA Licensing 102

Drug Discovery World

OCTOBER 4, 2023

The US Food and Drug Administration (FDA) has accepted Rocket Pharmaceuticals’ Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel). As the Principal Investigator in the US, I oversaw treatment of six of the nine LAD-I patients in this trial. In my opinion, the results are remarkable.

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Gene Therapy Gene Genetics Licensing 52

Drug Discovery World

MARCH 31, 2023

CSL’s Hemgenix (etranacogene dezaparvovec) has been authorised as the first gene therapy for adults with severe and moderately severe haemophilia B (congenital Factor IX (FIX) deficiency) in Great Britain. The post First gene therapy for haemophilia B approved in Great Britain appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Clinical Trials Clinical Trials 52

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

pharmaphorum

SEPTEMBER 13, 2021

AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease gene therapy appeared first on.

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Gene Therapy Gene Sales Trials 83

pharmaphorum

DECEMBER 16, 2022

Sio Gene Therapies’ litany of clinical disappointments over the last few years has finally led to the demise of the biotech, formerly known as Axovant and one of the ‘vant’ companies originally founded by entrepreneur Vivek Ramaswamy. ” The post Sio Gene Therapies comes to the end of its rocky road appeared first on.

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Gene Therapy Gene Licensing Licensing 57

Drug Discovery World

DECEMBER 2, 2022

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy. .

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

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Gene Therapy Gene Gene Expression Trials 52

Drug Discovery World

FEBRUARY 22, 2023

The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW). The treatment was approved by the US Food and Drug Administration in November 2022.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The post First gene therapy for haemophilia B gets go ahead in Europe appeared first on Drug Discovery World (DDW). World’s most expensive drug.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

NOVEMBER 23, 2022

uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy for adults with the bleeding disorder in the US. The post uniQure, CSL claim first okay for haemophilia B gene therapy appeared first on.

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Gene Therapy Gene FDA Approval Trials 57

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 first will be payable when a fifth patient has been given AK-OTOF in a phase I or phase 1/2 trial, as long as that happens before the end of 2024.

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Gene Therapy Gene Genetics FDA Approval 52

Pharmaceutical Technology

NOVEMBER 28, 2022

C4X Discovery (C4XD) and AstraZeneca have entered an exclusive global licensing agreement worth up to $402m to develop oral therapy to treat inflammatory and respiratory ailments. According to the deal, C4XD is entitled to get up to $16m in pre-clinical milestone payments before the commencement of the first clinical trial. .

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Development Gene Expression Licensing Licensing 264

pharmaphorum

OCTOBER 31, 2022

Actinium Pharma is on course to submit its targeted radiotherapy for acute myeloid leukaemia (AML) patients needing a bone marrow transplant in the US, buoyed by top-line data from a pivotal trial. The post Actinium eyes filing as conditioning drug for AML aces trial appeared first on.

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Trials Gene Therapy Drugs Antibody 112

pharmaphorum

FEBRUARY 8, 2022

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate AT845 hasn’t found any cause for concern. The post Astellas says Pompe gene therapy clears safety hurdle appeared first on.

Gene Therapy 52

Gene Therapy Gene Immune Response Licensing 52

Drug Discovery World

JANUARY 13, 2023

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment. . This is the largest and longest global Phase III study to date for any gene therapy in haemophilia, with 134 participants. .

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Gene Therapy Gene Licensing Licensing 52

Drug Discovery World

SEPTEMBER 16, 2022

Here, DDW Editor Reece Armstrong explores the global hubs of innovation for cell and gene therapies. Since the approval of Kymirah in 2017, the cell and gene therapy sector has emerged as an exciting and innovative market, representing some of the most promising drugs the pharmaceutical industry has ever seen. .

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Gene Therapy Gene Manufacturing Medicine 52

Pharmaceutical Technology

JUNE 23, 2022

The treatment is intended for patients with NSCLC harbouring alterations leading to mesenchymal-epithelial-transition factor gene (MET) exon 14 (METex14) skipping and for those who need systemic therapy after previous treatment with immunotherapy and/or platinum-based chemotherapy. It was discovered by Incyte and licensed to Novartis in 2009.

Medicine 263

Medicine Clinical Trials Clinical Trials Licensing 263

Camargo

OCTOBER 15, 2020

Often, to gain clinical trial design insights, a sponsor reviews the physician package inserts from approved New Drug Applications (NDAs) and Biologics License Applications (BLAs) with similar indications or in the same therapeutic area. What is the FDA’s current thinking on the use of single-arm trials?

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Production Trials Development Clinical Trials 190

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. On January 19, Editas announced that it entered into a definitive agreement with Shoreline Biosciences for the licensing of its natural killer cell program.

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Gene Therapy Drugs Gene Gene Editing 264

BioTech 365

OCTOBER 5, 2020

Investigational gene therapy licensed from The University of Manchester, UK aims to address both physical and CNS manifestations of Hunter syndrome Expands leading pipeline targeting lysosomal disorders with 5th investigational gene therapy Program expected to enter Phase 1/2 clinical trial … Continue reading →

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 29, 2021

US drugmaker Eli Lilly – still waiting for an FDA decision on one non-opioid pain drug – has just added another to its pipeline via a licensing agreement with Japan’s Asahi Kasei worth up to $410 million. The post Lilly adds to pain pipeline with Asahi Kasei licensing deal appeared first on.

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Licensing Licensing Bioavailability Antibody 52

Pharmaceutical Technology

JUNE 2, 2023

This is expected to streamline the path for ADCs to clinical trial and commercialisation. Lonza Licensing vice-president Ulrich Osswald said: “The Synaffix ADC technology is the gold standard, helping clinical-stage developers to design potentially curative therapies in areas of high unmet medical need.

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Licensing Licensing Gene Therapy Clinical Trials 130