gene therapy

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 …

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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 …

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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 …

Cancer concerns lead FDA to place clinical hold on BioMarin gene therapy Read More »

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 …

FDA slaps clinical hold on BioMarin’s PKU gene therapy Read More »

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 …

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

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” …

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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 …

Novartis cleared to restart trials of new Zolgensma delivery route Read More »

Ring Therapeutics rounds up $117M to find better viral vectors for gene therapy

The engineered viruses used to deliver gene therapies can spark complications and they can’t be re-dosed. Ring Therapeutics says viruses that evolved with humans can be better viral vectors and the startup has raised $117 million in new financing to continue its research.

Eye problems in more patients spells end of Adverum’s DME gene therapy program

Adverum Biotechnologies still doesn’t know if its experimental gene therapy caused eye problems reported in April in one patient in its diabetic macular edema study. But four others have since developed similar problems, and the company will no longer continue to develop the therapy for that indication.

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 …

NHS sets aside £340m for new Innovative Medicines Fund Read More »

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 …

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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 …

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INVEST Precision Medicine Conference Video: Biologics and the Supply Chain Ballet

This panel highlights how different organizations in the biopharma supply chain are addressing the inherent challenges of producing and delivering cell and gene therapies and biologics.

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 …

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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 …

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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 …

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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) …

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Biogen teams up with Ginkgo Bioworks in gene therapy manufacturing pact

Biogen is reaching across the Charles River, striking up a gene therapy manufacturing alliance with synthetic biology firm Ginkgo Bioworks. Ginkgo will use its technology to accelerate and make more efficient the process of manufacturing the AAV viral vectors used in gene therapies.

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 …

Charles River to Acquire Vigene Biosciences for $292.5M Read More »

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 …

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Andreessen Horowitz leads $100M Series A round in Dyno and its gene therapy tech

Dyno Therapeutics’ technology for designing viral vectors that deliver gene therapies has led to partnerships with Novartis, Sarepta Therapeutics, and Roche. With the Series A financing, the startup plans to expand its technology to address more tissue types, and potentially add more partners.

Gene therapy funding frenzy reaches Affinia as it reels in $110M in new capital

Affinia Therapeutics, which has technology that could deliver gene therapies to more tissue types in the body, raised the Series B financing as it looks ahead to clinical testing. The biotech is the latest company to close a substantial round of funding for gene therapy technology.

Capsida unveils $140M, AbbVie alliance & tech that takes gene therapy to the brain

Capsida Biotherapeutics’ technology can engineer viral vectors that deliver gene therapies to central nervous system cells. With that capability gave the startup was able to raise $50 million in Series A financing and a multi-drug research alliance AbbVie.

Adverum Bio’s shares plummet after patient in gene therapy study loses vision

Adverum Biotechnologies reported that a diabetic macular edema patient treated with the biotech’s experimental gene therapy developed complications, including blindness, in the treated eye. The company has unmasked the study and is trying to identify potential risks to other patients.

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 …

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FDA lifts clinical hold, clearing uniQure to resume hemophilia gene therapy trial

A uniQure gene therapy for hemophilia B that has been under a clinical hold for four months can now resume testing. The hold was lifted after uniQure answered the FDA’s questions about whether the gene therapy caused the liver cancer diagnosed in one patient who received the therapy.

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 …

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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 …

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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 …

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Bluebird analysis finds gene therapy ‘very unlikely’ to be cause of patient’s cancer

Bluebird bio said independent analyses found that the gene therapy inserted its payload at a site not known to cause cancer. Based on the results, the company has asked the FDA for permission to resume all of the clinical trials that were placed on hold in February during the inquiry.

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, …

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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 …

Gene and Cell Therapies in CNS Disorders: Miracle Cure? Opportunities Galore! Read More »

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 …

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Beam makes $120M bet that GuideTx’s tech brings gene editing beyond the liver

Base-editing biotech Beam Therapeutics engineered an all-stock buyout of Guide Therapeutics, a startup that finds, in vivo, the lipid nanoparticles that can deliver genetic medicines. Beam said GuideTx’s tech helps it address tissue types throughout the body, not just the liver.

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 …

Zynteglo halt re-ignites viral vector safety concerns; analysts Read More »

Novartis Collaborates with Bill & Melinda Gates Foundation to Discover and Develop Gene Therapy for Sickle Cell Disease

Shots: The Gates Foundation will provide funding support to discover and develop in vivo gene therapy for SCD. The alliance brings together Novartis drug discovery and gene therapy expertise with Gates’ funding’s The focus of the agreement to address the disparity in access to treatments and to prioritize populations and regions bearing the greatest burden …

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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 …

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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 …

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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 …

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Neurocrine nixes Parkinson’s gene therapy from Voyager Theraeutics alliance

The Neurocrine decision follows a clinical hold placed on the Parkinson’s program in December. It’s Voyager’s second loss of an R&D partner in the past year; AbbVie previously ended an alliance aiming to develop gene therapies for Parkinson’s and Alzheimer’s.

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 …

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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 …

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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 …

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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 …

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

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 …

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 Read More »

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 …

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‘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 …

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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 …

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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, …

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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 …

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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, …

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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 …

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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 …

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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 …

Bayer buys cell & gene therapy firm AskBio for $4 billion Read More »

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 …

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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 …

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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 …

EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD Read More »

Astellas’ Audentes Therapeutics discloses third patient death in gene therapy trial

The clinical trial had already been placed on hold in connection with the two prior patient deaths, which were linked to the higher dose of 300 trillion viral vectors per kilogram, while no deaths have been reported among those receiving the lower dose of 100 trillion vectors.

FDA rejects BioMarin’s hemophilia A gene therapy in ‘massive surprise’

In a CRL issued for valoctocogene roxaparvovec, the FDA told BioMarin that differences between its Phase I/II and Phase III studies limited the ability to rely on the earlier trial to support durability of effect, which an analyst said likely referred to a product comparability issue.

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 …

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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 …

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

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 …

Biogen plans trial of Spinraza in patients not responding to SMA gene therapy Read More »

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 …

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