The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy. Liver complications […]
What You Should Know: – FundamentalVR has raised an additional $20M to significantly accelerate medical skill-transfer and increase surgical proficiency through its world-leading medical simulation […]
Vector BioPharma has launched from the biotech startup incubator of Versant Ventures, which is backing the company with a $30 million Series A financing. Vector […]
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 […]
Shares in VBL Therapeutics have lost around 78% of their value in pre-market trading after the company reported a phase 3 trial of its lead […]
SAE Media Group’s 5th Annual Ophthalmic Drugs Conference 21 – 22 November 2022 | London, UK http://www.ophthalmicdrugs.com/PharmaPhorum Delving into advanced R&D and Delivery of Ocular […]
On this week’s pharmaphorum podcast, editor in chief Jonah Comstock catches up with Dr. Marie Wikström Lindholm, SVP of molecular design at Silence Therapeutics. mRNA is a […]
On this week’s pharmaphorum podcast, editor in chief Jonah Comstock catches up with Dr. Marie Wikström Lindholm, SVP of molecular design at Silence Therapeutics. mRNA is a […]
Gene therapy is making a world where cancer and AIDS can be cured, and a person can alter their genetic makeup, changing the direction of […]
New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have […]
SAE Media Group is delighted to announce the 2nd Annual Conference Oligonucleotide Therapeutics & Delivery, taking place on the 21st and 22nd September 2022 in […]
The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the […]
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 […]
bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its […]
The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far […]
Code Biotherapeutics uses synthetic DNA as the foundation for its genetic medicines, which the startup claims offer several key advantages compared to genetic medicines delivered […]
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 […]
A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way […]
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 […]
Cell and gene therapy research has been hot the past few years but the overall market has cooled down in recent months. During the World […]
What You Should Know: – Kelonia Therapeutics, a Boston-based biotech company focused on vivo gene delivery, launched with a $50 million in Series A funding […]
Pfizer now has the regulatory O.K. to resume pivotal tests of its gene therapy for Duchenne muscular dystrophy—but with new limitations and safety measures. The […]
Are you looking to solve challenges with current RNA therapeutics, such as the degradation of RNA by RNAses, the difficulty of RNA crossing the cell […]
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 […]
US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel […]
Evozyne uses artificial intelligence and machine learning to design novel proteins that can be used in gene therapies. The new agreement with Takeda Pharmaceutical covers […]
Gene therapy specialist bluebird bio has said 30% of its workforce will lose their jobs, in a bid to conserve cash as it tried to […]
Bayer is committing another €1.3 billion to Leaps by Bayer, the company’s investment arm, to support additional investments in companies developing innovative technologies in healthcare […]
Novartis is paying Voyager Therapeutics $53 million up front to begin a partnership focused on the discovery of capsids for central nervous system disorder gene […]
Voyager Therapeutics has signed another big pharma partner for its TRACER adeno-associated virus (AAV) vector technology, designed to improve the safety of gene therapies, with […]
As bluebird bio struggles to get its gene therapies over the finishing line, the company says its financial position raises “substantial doubt” about its ability […]
The SPAC merger that Amicus Therapeutics planned for its gene therapy assets has been called off due to market conditions. The rare disease company is […]
Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for […]
As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building […]
SpliceBio will use its €50 million in financing to advance development of a gene therapy for Stargardt disease, a rare eye disorder. The biotech’s technology […]
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 […]
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 […]
NHS England has agreed a confidential deal with Orchard Therapeutics that will allow access to Libmeldy – a gene therapy for a rare childhood disease […]
Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C>) from taking its place as an established treatment […]
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it […]
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 […]
The biggest biotech IPO of 2021 belongs to a cell therapy developer that has yet to reach the clinic. Cell therapy and artificial intelligence were […]
Novartis is acquiring gene therapy developer Gyroscope Therapeutics and its lead program, a potential treatment for the vision loss disorder geographic atrophy. It’s the latest […]
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 […]
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 […]
The FDA has halted tests of a Pfizer gene therapy for Duchenne muscular dystrophy. The clinical hold is one of several placed on 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 […]
AviadoBio’s gene therapies are designed to achieve widespread distribution throughout the central nervous system. The company’s lead program on track to begin human testing as […]
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) […]
Chroma Medicine is developing epigenetic therapies that turn genes on or off. The biotech startup has emerged from stealth mode backed by a $125 million […]
Sanofi is committing up to $60 million to Gyroscope Therapeutics, a clinical-stage biotech whose lead program could become the first gene therapy for a particular […]
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 […]
Shots: Genovis to receive up front for an exclusive license to Xork & is eligible to receive development & sales-based milestones along with royalties on […]
The progress of gene therapies continues to be stymied by safety risks associated with the viruses used to deliver them. Takeda Pharmaceutical is partnering with […]
The gene therapy pipeline of Voyager Therapeutics has hit several setbacks in the past year, but a strategy shift to its capsid technology is now […]
Amicus Therapeutics is spinning its gene therapy pipeline into a separate entity that will go public via a SPAC merger. Executives say the transaction puts […]
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 […]
Bioprocessing technologies company Repligen is acquiring Avitide, and its biologics purifying technology, in a $150 million cash and stock deal. Repligen said Avitide will help […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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. […]
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 […]
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 […]
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 […]
Experts in precision medicine provide a broad overview of where we are and where we need to go to make the promise and potential of […]
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 […]
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 […]
Cell and gene therapy companies already faced challenges in finding efficient ways to manufacture these new treatments. Then, the pandemic made these vulnerabilities even more […]
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 UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – […]
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 […]
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 […]
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 […]
A Biogen gene therapy for a rare, inherited eye disorder that leads to blindness has failed a key clinical trial. The preliminary results are the […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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! […]
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 […]
UniQure is making a case for lifting a clinical hold on its hemophilia B gene therapy, reporting Monday results of an independent investigation that appear […]
With the extension to its Series A financing, XyloCor plans a clinical trial testing its gene therapy as an adjunct to coronary artery bypass graft […]
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 […]
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, […]
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 […]
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 […]
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 […]
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. […]
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 […]
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 […]
Cancer is a known risk of lentiviral-based gene therapies. Though bluebird says its gene therapy is engineered to avoid that risk, the company is suspending […]
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 […]
Ensoma contends its technology avoids the complexities of other approaches to gene therapy, which could expand genomic medicine’s reach to more patients and more diseases. […]
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 […]
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 […]
Sana Biotechnology is turning new science into novel medicines intended to overcome limitations of current cell therapies. After going from stealth to IPO in two […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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. […]
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 […]
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 […]
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 clinical trial had already been placed on hold in connection with the two prior patient deaths, which were linked to the higher dose of […]
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 […]
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 […]
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 […]
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 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 […]
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