Category: gene therapy

  • Sarepta says early filing for DMD gene therapy may be back on

    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. The new data from the mid-stage Study 102 of SRP-9001 (delandistrogene moxeparvovec) comes from 21 patients who were initially […]

  • Avrobio shifts gene therapy priorities as data surprise shelves lead program

    Avrobio is stopping enrollment in a clinical trial for its Fabry disease program after new results showed variability in how the gene therapy was taken up by patients. No safety problems were reported, but the biotech said it now plans to focus its other gene therapy candidates for rare metabolic disorders.

  • Novartis adds gene therapy for blindness with Gyroscope buy

    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. Just a few weeks France’s Sanofi made a $40 million investment in Gyroscope for first refusal rights on the gene therapy – called GT005 – […]

  • Pfizer pauses Duchenne gene therapy trial after patient death

    Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. In an update posted today the pharma giant said “we do not yet have complete information and are actively working with the trial site investigator to understand […]

  • Trial sets up filings for uniQure’s haemophilia gene therapy

    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 results are from the HOPE-B study, which was temporarily placed on a clinical hold by the FDA earlier this year […]

  • Dyno attracts $1.6bn gene therapy deal with Astellas

    Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. The Japanese drugmaker is paying $18 million upfront to exercise an option on the AAV platform – which my help […]

  • Sanofi winds $40m into UK gene therapy firm Gyroscope

    French drugmaker Sanofi has made a $40 million investment in Gyroscope Therapeutics, with another $20 million potentially on offer as the UK biotech advances its clinical-stage gene therapies for ophthalmic diseases. The investment gives Sanofi rights of first refusal on GT005, Gyrocope’s lead gene therapy for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), […]

  • Selecta Enters into an Exclusive License Agreement with Genovis to Advance IgG Protease in Gene Therapy and Autoimmune Disease

    Shots: Genovis to receive up front for an exclusive license to Xork & is eligible to receive development & sales-based milestones along with royalties on sales globally & holds the rights for research, preclinical, diagnostic & other non-therapeutic applications of Xork The collaboration will utilize Genovis’ IgG protease, IdeXork (Xork & Selecta’s ImmTOR platform for […]

  • Aiming for better genetic medicines delivery, startup GenEdit grabs $26M

    Biotech startup GenEdit is developing polymer nanoparticle technology to deliver genetic medicines, an approach intended to avoid the limitations of viral vectors. Already partnered with a clinical-stage company, it now has financial support from a big pharma giant that joined a syndicate of investors in a $26 million Series A round.

  • Astellas Pharma reports fourth patient death in gene therapy clinical trial

    The death occurred less than two weeks after Astellas Pharma reported that the patient developed liver problems after being dosed with the experimental gene therapy for a rare neuromuscular disorder. Astellas said the cause of death is still under investigation, and the FDA has placed a clinical hold on the study.

  • Astellas reports fourth death in halted gene therapy trial

    There has been a fourth patient fatality in Astellas’ clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to safety concerns. The latest suspension came after abnormal liver function tests (LFTs) were seen in a boy receiving the gene therapy, who has since […]

  • AbbVie spies potential in Regenxbio eye disease gene therapy

    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 partnership centres on RGX314, which delivers a gene coding for an antibody against VEGF – a well-established approach to treating […]

  • Cancer concerns lead FDA to place clinical hold on BioMarin gene therapy

    A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed […]

  • FDA slaps clinical hold on BioMarin’s PKU gene therapy

    BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The biotech said that the US regulator ordered the pause on the study after liver tumours were […]

  • More trouble for Astellas as AT132 gene therapy trial is halted again

    Astellas has halted dosing in a study of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM) for the second time after another serious adverse event (SAE) linked to possible liver damage. The ASPIRO trial of AT132 has been paused after abnormal liver function tests (LFTs) were seen in a patient receiving […]

  • Gene therapy specialist bluebird exits “untenable” European market

    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. On the firm’s second-quarter results call, Obenshain said the biotech will start “an orderly wind-down” […]

  • Novartis cleared to restart trials of new Zolgensma delivery route

    The FDA has relaxed a clinical hold on clinical trials of Novartis’ intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get underway. Zolgensma (onasemnogene abeparvovec) is already approved as an intravenous treatment in babies and very young children for the rare muscle-wasting disease, but the new […]

  • NHS sets aside £340m for new Innovative Medicines Fund

    NHS England has matched its financial backing of the Cancer Drugs Fund (CDF) with an equivalent pot of money for a new Innovative Medicines Fund (IMF) that will provide early access to cutting-edge medicines like cell and gene therapies. It has proposed that the IMF will work in the same way as the CDF, funding […]

  • Bluebird claims EU nod for rare disease therapy Skysona

    Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The EMA has cleared Skysona (elivaldogene autotemcel) for use in CALD patients aged under 18 who have an ABCD1 genetic mutation and no matched […]

  • NICE rejects Orchard’s gene therapy for rare childhood disease MLD

    Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there was evidence of a short-term benefit with Libmeldy (atidarsagene autotemcel or OTL-200), assumptions about its long-term effects are uncertain, making it unclear whether it will offer […]

  • Biogen’s ambitions in gene therapy hit by another failed pivotal trial

    When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category.  Just over two years later, both of those candidates have failed late-stage clinical trials, leaving Biogen’s $800 million investment in Nightstar looking like a poor deal. The […]

  • Bayer’s two-pronged cell/gene therapy assault on Parkinson’s enters clinic

    Bayer subsidiary BlueRock Therapeutics has dosed the first Parkinson’s disease patient in a phase 1 trial of its cell-based therapy DA01, which aims to replenish the neurons lost in people with the neurodegenerative disorder. At the same time, the German group’s Asklepios BioPharma (AskBio) unit has started recruiting patients for a phase 1b trial of […]

  • New Cell & Gene Therapy Collective seeks to boost UK adoption

    The UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – but there are still many barriers to wider adoption. These challenges often arise because these therapies go through the same value assessment processes as less complex medicines, and the infrastructure […]

  • CHMP backs bluebird bio’s gene therapy for rare disease ALD

    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. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has given the go-ahead to bluebird’s therapy – known as Skysona (elivaldogene autotemcel) […]

  • Charles River to Acquire Vigene Biosciences for $292.5M

    Shots: Charles River Laboratories to acquire Vigene in all cash transaction for $292.5M. In addition to the initial purchase price, the transaction includes contingent additional payments of up to $57.5M based on future performance The transaction is expected to be close in Q3’21. The acquisition will allow Charles River to boost its gene therapy capabilities […]

  • First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

    The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Pfizer’s CIFFREO study began in the US in January this year and has now begun enrolling eligible patients in the UK. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one […]

  • FDA lifts hold on uniQure gene therapy after cancer case review

    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 biotech said that the FDA had concluded that its investigation into the case, conducted in collaboration […]

  • 2nd Gene Therapy for Neurological Disorders Europe

    Returning digitally in 2021, the 2nd Annual Gene Therapy for Neurological Disorders Europe meeting will possibly be your last chance to attend a European meeting without the airmiles! Focusing on the industry’s challenges of developing and delivering gene therapies to the CNS and bringing fresh faces and emerging companies into the conversation, this is not one to miss […]

  • 2nd Gene Therapy Immunogenicity

    The 2nd Gene Therapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your gene therapy candidate. In the context of ever-increasing gene therapy clinical activity and uncertainty around immunogenicity challenges, the Gene Therapy Immunogenicity Summit will unite large pharma and innovative biotechs to ensure that you’re up to speed on […]

  • bluebird bio announces long-term data from CALD gene therapy

    bluebird bio has announced new clinical trial data from its gene therapy for patients with cerebral adrenoleukodystrophy (CALD), including long-term follow-up data. CALD is the most severe form of adrenoleukodystrophy (ALD), a rare, X-linked metabolic disease affecting 1 in 21,000 newborn boys worldwide. Approximately 40% of boys with ALD will develop CALD1, typically between the […]

  • bluebird seeks gene therapy trial restart after cancer scare

    bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, […]

  • Gene and Cell Therapies in CNS Disorders: Miracle Cure? Opportunities Galore!

    CNS disorders are a wide-ranging set of disorders in which the brain loses its normal functioning, limiting everyday ability to function. These may arise from various reasons-some are inherited, some result from damage, and some from infections. It contributes to the highest disability-adjusted life years. The current treatment options are minimal. Most of the disorders […]

  • Sanofi Collaborates with Sirion to Develop Gene Therapy Treatment with Improved AAV Capsids

    Shots: The companies collaborated to develop improved tissue-selective AAV vectors to realize effective gene therapy treatments for multiple life-threatening disorders The focus of this agreement is to develop new and modified AAV capsids that exhibit a safe product profile with improved specificity and higher gene delivery efficiency The collaboration comes two years after Sirion signed […]

  • Zynteglo halt re-ignites viral vector safety concerns; analysts

    Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface.  That’s the view of analysts at Jefferies, who suggest it could “re-ignite concerns over the […]

  • bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

    It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. NICE is assessing Zynteglo (betibeglogene autotemcel), a one-off gene therapy for the condition, which can have life-threatening consequences and is associated with a curtailed […]

  • Richard Francis on his Purespring plans: the pharmaphorum podcast

    In this episode of the pharmaphorum podcast, our news editor Richard Staines talks to Richard Francis, who took over as CEO of gene therapy company Purespring Therapeutics earlier this month. Francis was previously CEO of Sandoz, Novartis’ generics and biosimilars division, where he led the development of biosimilars and also sat on the Swiss pharma […]

  • Why Cancer Registries are Part of America’s Fight for Racial Equality

    Warren A Whyte, Ph.D., VP Scientific Partnerships at ConcertAI According to a 2019 study in the Proceedings of the National Academy of Sciences, one in 1,000 Black males can expect to die at the hands of the police. Black males were also 2.5 times more likely to die during an encounter with police than white […]

  • Gene editing: beyond the hype

    Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. ‘Cutting edge’ is, for once, a truly […]

  • Gene Therapy Comparability

    The inaugural Gene Therapy Comparability Summit is the only industry dedicated meeting to ensuring your manufacturing processes are effective and comparable at every stage of development, supporting a seamless transition to the larger scales required for commercialization. This meeting will unite large pharma and innovative biotechs including Pfizer, Biogen, BioMarin and Takeda as they share best practices, lessons learned and analytical […]

  • Gene Therapy for Muscular Disorders

    With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. Despite the successes, there are still withstanding challenges for gene therapy developers targeting the muscle when it comes to optimizing delivery and […]

  • Biohaven’s Troriluzole Failure; Daiichi/ AZ’s Enhertu; Fujifilm & Manufacturing Spree; J&J’s Darzalex Faspro

    Biohaven’s Troriluzole Dwindles Again In Alzheimer’s After Anxiety  Biohaven Pharmaceuticals had put too much faith in its third-generation prodrug, Troriluzole. The company has tested the efficacy of the drug in more than one indication, including generalized anxiety disorder (GAD), obsessive-compulsive disorder, spinocerebellar ataxia and Alzheimer’s disease (AD). However, it seems the drug continues to disappoint […]

  • Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

    Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor […]

  • Rocket Pharmaceuticals in orbit after gene therapy read-out

    Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease. Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than […]

  • ‘Knowledge is power’ for rare diseases and NAbs

    Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. Neutralising antibodies (NAbs) present a unique challenge to researchers looking to treat patients with AAV treatment. As AAV is a virus derived from the common cold, the immune system can easily […]

  • 2nd Gene Therapy for Blood Disorders

    2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their beta-thalassemia candidate. With pipelines advancing and emerging companies joining the […]

  • Gene Therapy Medical Affairs

    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. Industry pioneers from leading pharma and biotech companies at various stages of development and commercialization including Novartis Gene Therapy, Orchard Therapeutics & Bluebird Bio will share how to engage effectively with internal clinical and commercial teams as well as external HCPs, […]

  • Helixmith starts new trial of diabetic neuropathy gene therapy

    Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study. The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida. The aim is […]

  • UCB Acquires Handl Therapeutics to Augment its Gene Therapy Portfolio

    Shots: The acquisition will bolster UCB’s pipeline program, capabilities, and platforms in the gene therapy space. The Handl Therapeutics will continue to be based in Leuven, Belgium while working closely with UCB’s international research teams In addition, the UCB collaborated with Lacerta to focus on CNS diseases, under which Lacera will lead research, preclinical activities, […]

  • UCB revs up its gene therapy drive with Handl acquisition

    UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The mid-sized pharma group has also signed a collaboration with Florida, US-based Lacerta Therapeutics, adding more AAV capsids – well established […]

  • Novartis keeps a close eye on gene therapy with Vedere Bio buy

    Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Incubated by Atlas Ventures, Cambridge, Massachusetts Vedere has been flying under the radar since it was founded in 2019 to develop adeno-associated […]

  • Bayer paying up to $4B for gene therapy pioneer AskBio

    The deal, offering $2 billion up front and $2 billion in future milestone payments, includes a gene therapy manufacturing facility and a pipeline that includes treatments for Parkinson’s and congestive heart failure.

  • Bayer buys cell & gene therapy firm AskBio for $4 billion

    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. AskBio’s portfolio includes pre-clinical and clinical stage candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases. Through the acquisition, Bayer will gain full rights to the […]

  • Cleveland Clinic Names Top 10 Medical Innovations For 2021

    Cleveland Clinic’s top clinicians and researchers present the top 10 medical innovations transforming medical advancements and new awards to recognize healthcare innovation. The list of breakthrough technologies was selected by a committee of Cleveland Clinic subject matter experts, led by Will Morris, M.D., executive medical director for Cleveland Clinic Innovations, and Akhil Saklecha, M.D., managing […]

  • Axovant pushes on with Parkinson’s gene therapy, despite investor cynicism

    Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. However shares in Axovant were down sharply after the announcement because of market sentiment that the New York biotech had been selective with the data it had […]

  • EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

    Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD […]

  • Preparing for an influx of cell and gene therapy approvals

    Cell and gene therapies offer some of the most groundbreaking advancements in patient care the pharma industry has ever seen. However, to fully realise the potential of these innovative therapies, integration across the supply chain is critical – particularly with reimbursement and logistics. As of the end of 2019, there were 17 cell and gene […]

  • Kite’s Tecartus (brexucabtagene autoleucel) Receives the US FDA’s Approval as the First Cell-Based Gene Therapy for Relapsed or Refractory MCL

    Shots: The accelerated approval follows FDA’s PR and BT designation and is based on ZUMA-2 study assessing Tecartus (formerly KTE-X19) in 74 patients with r/r MCL prior treated with  anthracycline/ bendamustine-containing CT, an anti-CD20 Ab therapy and a BTK inhibitor (ibrutinib or acalabrutinib) Results: 87% patients responded to Tecartus (single infusion), including 62 % patients […]

  • Biogen plans trial of Spinraza in patients not responding to SMA gene therapy

    Biogen is planning to trial its Spinraza spinal muscular atrophy (SMA) drug in patients who have not responded properly to Novartis’ gene therapy Zolgensma, combining the two ultra-expensive therapies to treat the rare muscle-wasting disease. In the US, a single shot of Zolgensma (onasemnogene abeparvovec) costs $2.1 million, while Spinraza (nusinersen) costs $750,000 in its […]

  • Pandemic hits sales at Novartis after strong launch for SMA gene therapy

    The COVID-19 pandemic has bit into sales at Novartis, the big Swiss pharma said in second quarter results, after orders fell back following a period of stockpiling earlier in the year. Figures for the quarter showed reported sales fell by 4% to $11.3 billion compared with the corresponding period last year, although favourable currency fluctuations […]