Japanese Government Panel Oks Expensive Gene Therapy for Rare Disease
February 26, 2020 – (Japan Times) – A health ministry panel approved Zolgensma, a gene therapy for the treatment of spinal muscular atrophy. The health minister is expected to formally approve Zolgensma by the end of March, with public health insurance coverage seen starting this summer.
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New Report from ARM Provides Overview of the Regenerative Medicine Landscape for Rare Disease
February 24, 2020 – (ARM) – ARM Regenerative Medicine & Rare Disease 2019 report provides updated metrics and trends for the 420 regenerative medicine companies globally developing therapies for rare diseases. ARM reports that there are 647 ongoing rare disease clinical trials and that developers in this space raised $6.4B in 2019.
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BioMarin Talks up Potential Savings as Hemophilia Gene Therapy Launch Looms
February 27, 2020 – (Endpoints News) – BioMarin's COO has suggested that the company is open to the kind of payment over time model that Novartis has adopted (payers spread the cost over 5 years after the one-time injection), or an outcomes-based model where they only get paid if the therapy continues to work, a plan bluebird is testing in Europe.
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Iovance Biotherapeutics Explores a Potential Sale
February 25, 2020 – (Bloomberg) – Cancer drugmaker Iovance Biotherapeutics is exploring a sale and has held preliminary talks with potential buyers. No final decision has been made and Iovance could opt to remain independent.
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More Cell and Gene Therapy Facilities in the Hundreds are Needed
February 25, 2020 – (Genetic Engineering and Biotechnology News) – The bioindustry will require more cell and gene therapy plants, says an expert in the industry. The recent surge in clinical activity in regenerative medicines has not been matched by an increase in production capacity for cell and gene therapies.
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Trakcel and Ori Biotech Sign a Collaboration Agreement for Supply Chain Orchestration Within Cell and Gene Therapy Manufacturing
February 25, 2020 – (Trakcel) – TrakCel and Ori Biotech announced a non-exclusive strategic partnership to achieve supply chain orchestration within closed system manufacturing for the cell and gene therapy sector.
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Axovant Announces Closing of $74.7M Public Offering of Common Shares and Prefunded Warrants, Including Full Exercise of Underwriters’ Option to Purchase Additional Shares
February 24, 2020 – (Axovant Gene Therapies) – The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, and Parkinson’s disease.
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Fortress Biotech Announces Closing of Series A Preferred Stock Offering and Full Exercise of Over-Allotment Option
February 24, 2020 – (Fortress Biotech) – Fortress has received aggregate gross proceeds, before deducting underwriting discounts and commissions and offering expenses, of approximately $14.4M. Fortress intends to use the net proceeds from the public offering for its operations and general corporate purposes.
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Immatics and GSK Partner to Develop Novel Adoptive Cell Therapies
February 20, 2020 – (Immatics) – Under the terms of the agreement, Immatics will receive an upfront payment of €45M ($50M) for two initial programs and is eligible to receive over $550M in development, regulatory and commercial milestone payments for each product.
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Incysus Therapeutics Initiates Enrollment in Phase 1 Study of a Genetically Modified Gamma-Delta T Cell Immunotherapy in Patients with Newly Diagnosed Glioblastoma
February 27, 2020 – (Incysus Therapeutics) – Incysus’ DRI approach seeks to combine conventional chemotherapies with a gamma-delta T cell based immunotherapy to modify the tumor microenvironment and drive the immune system.
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Tessa Therapeutics Announces FDA RMAT Designation Granted to its CD30 CAR-T Cell Therapy for the Treatment of r/r CD30-positive Classical Hodgkin Lymphoma
February 27, 2020 – (Tessa Therapeutics) – The RMAT designation is supported by clinical data from two independent CD30 CAR-T Phase I/II studies. Both studies demonstrated objective response rates of more than 70%, with 18 patients achieving complete response out of 27 patients treated with CD 30 CAR- T with lymphodepleting chemotherapy as of November 2019.
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Kiadis Pharma Announces FDA Clearance of Clinical Study in r/r AML With Off-The-Shelf NK Cells From Universal Donors
February 26, 2020 – (Kiadis Pharma) – The trial is expected to beginning enrolling patients in March. The NK cells are not genetically engineered, which eliminates the need for long-term follow up of patients, and reduces the overall regulatory burden.
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Biogen Touts New Evidence From the Gene Therapy Company It Wagered $800M On
February 25, 2020 – (Endpoints News) – Biogen reported that all 18 patients tolerated their retinal gene therapy and had dose-dependent responses. All patients were back to baseline vision levels 3 months after the procedure, and 7 of them had improved vision through 6 months of follow-up.
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Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA
February 25, 2020 – (Lysogene) – LYS-SAF302 has previously received Orphan Drug Designations for the treatment of MPS IIIA in the European Union in 2014 and in the US in 2015, as well as Rare Pediatric Disease Designation in the US. The therapy is being investigated in an international Phase 2/3 trial.
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China's NMPA Will Review New Drug Application for Yescarta for the Treatment of Certain R/R B Cell Lymphomas
February 24, 2020 – (Fosun Pharma) – Fosun Kite Biotechnology has received the acceptance notice for Axicabtagene Ciloleucel (Yescarta) from the Chinese NMPA for review of its marketing registration. The NMP will review Yescarta for the treatment of adult patients with relapsed and refractory large B-cell lymphoma (including DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
- A CAR-T Race Intensifies (February 25, 2020 – STAT News) – In addition to the NDA for Yescarta, the NMPA disclosed recently that it has given the go-ahead to Novartis’ clinical trial application for Yescarta.
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Magenta Therapeutics Completes Dosing in Phase 1 MGTA-145 Trial, Demonstrating Rapid, Single-Day First Line Stem Cell Mobilization and Collection
February 24, 2020 – (Magenta Therapeutics) – Magenta has completed dosing in this Phase 1 trial, achieving all primary and secondary endpoints. The Company plans to move the MGTA-145 program into multiple Phase 2 studies in 2020.
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Mesoblast Announces Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GvHD
February 24, 2020 – (Mesoblast) – In the aggregated dataset, 204 of the 309 (66%) patients achieved an overall response at Day 28 following a four-week course of Ryoncil. Results were consistent across all grades of disease, including the most severe.
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ReNeuron Announces Positive Long-Term Data in Retinal Clinical Trial
February 24, 2020 – (ReNeuron) – Data twelve months post-treatment showed sustained, clinically relevant improvements in visual acuity compared with baseline, as measured by the number of letters read on the ETDRS chart.
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New CRISPR Base-Editing Technology Slows ALS Progression in Mice
February 26, 2020 – (University of Illinois at Urbana-Champaign) – By combiningAAV gene delivery and split-base editors, researchers targeted and permanently disabled a mutant SOD1 gene, which is responsible for roughly 20% of inherited forms of ALS.
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Curing Genetic Disease in Human Stem Cells
February 20, 2020 – (Hubrecht Institute) – Scientists used base-editing for the first time to show that CRISPR can safely cure cystic fibrosis in human stem cells.
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Plant-Based Relatives of Cholesterol Could Give Boost to Gene Therapy
February 20, 2020 – (Oregon State University) – Scientists found that gene-infused nanoparticles used in gene therapy work better when they include plant-based relatives of cholesterol. The shape and structure helped the genes reach the cytosol in the cell, where the delivered genes can perform their intended function.
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Hahn, FDA Center Directors Discuss What’s Necessary to Better Address Rare Diseases
February 24, 2020 – (Regulatory Focus) – FDA Commissioner Stephen Hahn discussed at a public forum the importance of driving rare disease drug development by unleashing the data from very small populations. He addressed the need to integrate more data sources to modernize FDA’s approach and stressed the importance of the patient voice.
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MEPs Claudia Gamon (Renew Europe) and Tomislav Sokol (EPP) Co-Host Advanced Therapies Policy Breakfast, Organized by EuropaBio and ARM
February 20, 2020 – (EuropaBio) – On February 19, EuropaBio and the Alliance for Regenerative Medicines (ARM) organised a debate on the potential of advanced therapies for currently incurable diseases. The event, co-hosted by MEPs Claudia Gamon (Renew Europe) and Tomislav Sokol (EPP), discussed strategies to promote patient access to advanced therapies in Europe.
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If DNA Is Like Software, Can We Just Fix the Code?
February 26, 2020 – (MIT Tech Review) – One serious challenge facing gene drugs for individuals is that short of a healing miracle, it may ultimately be impossible to be sure they really work. That’s because the speed with which diseases like A-T progress can vary widely from person to person. Proving a drug is effective, or revealing that it’s a dud, almost always requires collecting data from many patients, not just one.
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OPINION: Should ‘Broken’ Genes Be Fixed? My Daughter Changed the Way I Think About That Question
February 21, 2020 – (STAT News) – Ruthie’s presence in this world makes it a better, kinder, more considerate, more patient, and more humane place. It is not hard, then, to see that these new technologies bring risk that the world will be less kind, less compassionate, and less patient when there are fewer children like Ruthie.
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Vice President, Global Policy and Advocacy
Alliance for Regenerative Medicine – Washington, DC
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