Trend Chart On Innovative Bioindustries – June 2020, 2nd

Trend Chart On Innovative Bioindustries – June 2020, 2nd

Trend Chart On Innovative Bioindustries – June 2020, 2nd


Novartis snags EU nod for SMA gene therapy
After a manufacturing-related delay, Novartis has won conditional approval in the EU for Zolgensma®, the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and others with up to three copies of the SMN2 backup gene. Zolgensma® has demonstrated significant and clinically meaningful therapeutic benefit in pre-symptomatic and symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease and to date, sustained for 5 years post-dosing. Immediate access to Zolgensma®, aligned to the label, is available in France through the ATU framework and expected shortly in Germany. Data from the Phase 3 STR1VE trial show prolonged event-free survival, increases in motor function and significant milestone achievement in patients with SMA Type 1, consistent with the Phase 1 START trial. AveXis in discussions with EU governments and reimbursement agencies to agree on Zolgensma®’s EU label is different from the U.S. version. While it’s approved by the FDA to treat children less than 2 years of age, the EMA allows it in babies and young children who weigh up to 21 kilograms. According to a Pediatric Neuromuscular Clinical Research natural history study of SMA, almost all patients under the age of 5 will be under 21 kg. For further information

Related Informations/Publications:
Ann Pharmacother. 2020 Mar 23. “Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy“. Stevens et al. Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.
Results / Comments: Onasemnogene appears to be an efficacious therapy for younger pediatric patients with SMA type 1. Concerns include drug cost and potential liver toxicity. Long-term benefits and risks have not been determined. Link: Abstract
MAR 2020: AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA). Link: Press Release
MAY 2019: AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA). Link: Press Release . You can also click here to get full-page newspaper articles.


Using MHcut and commercial genome-editing technology, researchers can create mutations in iPS cells with extraordinary precision to model diseases without the need of patient samples. ©Kyoto University/Woltjen lab● Precision Therapy for inherited retinal disease: at the forefront of genomic medicine
Inherited retinal diseases (IRDs) represent a diverse array of conditions characterized by dysfunction or loss of 1 or more retinal cell types. Next-generation sequencing has enabled rapid and relatively inexpensive genotyping, with more than 250 genes identified as responsible for IRDs. This expansion in molecular diagnostic accuracy, in combination with the retina’s relative accessibility and immune privilege, has fostered the development of precision therapies to treat these myriad conditions. Novel techniques are being used in early trials. Precision molecular therapies for IRDs hold great promise as diagnostic and treatment strategies continue to expand. The review appeared in the June issue of Clin Lab Med.

Related Informations / Publications
-Expert Opin Biol Ther. 2020 Jun;20(6):565-578. Voretigene Neparvovec-Rzyl for Treatment of RPE65-mediated Inherited Retinal Diseases: A Model for Ocular Gene Therapy Development. Ciulla TA et al. Indiana University School of Medicine, Indianapolis, IN, USA. Link: Abstract
Int J Mol Sci. 2020 Mar 27;21(7):2329. CRISPR Interference-Potential Application in Retinal Disease. Peddle CF et al. University of Oxford, Oxford OX3 9DU
Results / Comments: This review details how CRISPR interference might be used to treat retinal diseases and addresses potential challenges for clinical translation. Link: Abstract
Clin Ophthalmol. 2020 Mar 5;14:707-719. The Impact of Inherited Retinal Diseases in the Republic of Ireland (ROI) and the United Kingdom (UK) From a Cost-of-Illness Perspective. Galvin O et al. Retina International, Dublin 8, D08 R9CN, Ireland
Results / Comments: This review reveals that IRD patients do not frequently engage the healthcare system and as such suggests a cost-of-illness model from a societal perspective may be a better format. Link: Abstract
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©National-Institutes-of-Health-NIH-via-VisualHunt● AAV-Based Gene Therapy Products: Ongoing Clinical Trials
BioPharmAnalyses and OctopusyX BioConsulting are proud to announce the launch of their latest report:”AAV-based gene therapy Products: Ongoing Clinical Trials“. This 205-page report provides you with a unique exhaustive overview of 116 ongoing trials for the treatment of 40 diseases (as of June 2020). More than 80 products from 49 companies and academic institutions are described.
Areas covered: Cardiovascular Diseases, Hematological Diseases, Hepatic Diseases, Infectious Diseases, inherited Metabolic Diseases, Lysosomal Diseases, Neurological Diseases, Neuromuscular Diseases and Ophthalmological Diseases
You can click here to get sample pages and click here to get the order form (€590, excl. VAT)


● Cancer cell therapies: the clinical trial landscape
Cell therapies constitute the largest number of agents in development in immuno-oncolog. In a recent review, American researchers provide an update on the pipeline and clinical trials of cancer cell therapies. They also compare the current landscape (with a March 2020 data cut-off point) with our previous update from March 2019). The current global cancer cell therapy pipeline includes 1,483 active agents, 472 more than last year. Among the different cell therapy types, the chimeric antigen receptor (CAR)-T cell class has the largest increase (290 agents this year versus 164 in 2019), whereas novel T cell approaches (such as CRISPR engineered T cells or γδT cells) and other cell therapies (such as macrophage-based therapies) have increased to 49 and 56 agents, respectively. To improve our understanding of the year-on-year developments, the active agents were reclassified based on their origin as autologous or allogeneic (off-the-shelf). A majority of cellular immunotherapies (667) in development are autologous in nature. However, the greatest percentage increase from those reported last year comes from preclinical (73.8% increase) and phase I (90.9% increase) development of allogeneic therapies. For most therapies in phase II and beyond that are being developed in countries other than the United States, it has not been disclosed whether they are autologous or allogeneic. Of note, the previously marketed allogeneic agent nalotimagene carmaleucel was withdrawn from the EU markets by its manufacturer MolMed in October 2019 following its failure to improve disease-free survival in a phase III trial. The review appeared in May 26th online issue of Nat Rev Drug Discov

Related Informations / Publications
Lab Chip. 2020 May 27. Machine Learning-Aided Quantification of Antibody-Based Cancer Immunotherapy by Natural Killer Cells in Microfluidic Droplets. Sarkar S et al. Northeastern University, 360 Huntington Avenue, Boston, MA, USA
Results / Comments: The results suggest that this semi-automated single cell assay can reveal the variability and functional potency of NK cells and may be used to optimize immunotherapeutic efficacy for preclinical analyses. Link: Abstract
Int J Mol Sci. 2020 May 21;21(10):E3650. The Role of Immune Checkpoints After Cellular Therapy. Schmitz F et al. University Hospital Bonn, 53127 Bonn, Germany. Link: Abstract
Biochem Pharmacol. 2020 May 21:114051. Emerging CAR Landscape for Cancer Immunotherapy. Lim FLWI et al. Singapore General Hospital Outram Road, 169608 Singapore Singapore. Link: Abstract . You can also click here to get full-page newspaper articles. 

● IRBM’s Advent announces manufacture of 13,000 doses of Covid-19 vaccine
Advent, an IRBM company and global leader in viral vector manufacturing, announced on May 28th that it has manufactured 13,000 doses of the novel Covid-19 vaccine candidate, ChAdOx1 nCoV-19 (now known as AZD1222), the former ChAdOx1 nCoV-1 is a replication-deficient simian adenoviral vector). The first 4,000 doses have been delivered for use in the Phase 2/3 clinical trial COV002 by the University of Oxford, England. Following a long history of partnership, including the announcement on 7 February of an agreement between The Jenner Institute and IRBM’s Advent to manufacture ChAdOx1 nCoV-19, Advent has now manufactured its first batch of vaccine in just over one month from receiving the seed stock, whilst ensuring high standards of quality control. Over the past decade, Advent has manufactured hundreds of thousands of doses of vaccine for over 10 different infectious diseases. The company has achieved a 100% success rate for the approval of clinical grade quality batches by regulatory authorities. Advent rapidly devised a manufacturing process incorporating high quality process development, and extensive quality control, to produce 13,000 doses of the vaccine for use in Phase 2/3 clinical trials. With Phase I trials already enrolled for this vaccine, rapid and reliable development of a large scale manufacturing process is a critical step in quickly and safely delivering the vaccine for further trials. On the other hand, Oxford Biomedica plc announced on May 28th that it has signed a one year Clinical & Commercial Supply Agreement with AstraZeneca. The agreement relates to the GMP manufacture of the adenovirus vector based COVID-19 vaccine candidate, AZD1222, which recently entered clinical trials at multiple sites in the UK. Oxford Biomedica is working alongside AstraZeneca and other manufacturing organisations to provide large scale manufacturing capacity for this vaccine candidate. For further information, see B3C newswire and Reuters .
Picture caption: Ultrastructural morphology exhibited by coronaviruses similar to COVID-19 which became pandemic. © Public Health Image Library, Centers for Disease Control and Prevention,USA.

Related Informations / Publications
J Immunother Cancer. 2020 May;8(1):e000933. COVID-19 and Immune Checkpoint Inhibitors: Initial Considerations. Sullivan RJ et al. Massachusetts General Hospital Cancer Center, Boston, Massachusetts, USA. Link: Abstract
-FEB 2020: The Jenner Institute signs an agreement with Advent to develop a novel coronavirus vaccine Link: Press Release
SEP 2019: IRBM signs agreement with MD Anderson Cancer Center to generate novel immune-checkpoint monoclonal antibodies. Link: Press Release
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Using MHcut and commercial genome-editing technology, researchers can create mutations in iPS cells with extraordinary precision to model diseases without the need of patient samples. ©Kyoto University/Woltjen lab● Fate Therapeutics announces FDA clearance of IND application for FT538, first CRISPR-edited, iPSC-derived cell therapy
Fate Therapeutics, a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced on May 20th that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for FT538, the first CRISPR-edited, iPSC-derived cell therapy. FT538 is an off-the-shelf natural killer (NK) cell cancer immunotherapy that is derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components to enhance innate immunity: a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor; an IL-15/IL-15 receptor fusion (IL-15RF); and the elimination of CD38 expression. The Company plans to initiate clinical investigation of three once-weekly doses of FT538 as a monotherapy in acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-directed monoclonal antibody therapy, for the treatment of multiple myeloma. For further information

Related Informations / Publications
APR 2020: Fate Therapeutics Announces Worldwide Collaboration with Janssen for Novel iPSC-derived Cell-based Cancer Immunotherapies
Results / Comments: Fate to receive $50 million upfront payment and $50 million equity investment, plus full funding for the research and development of collaboration candidates through IND filing. Link: Press Release
-J Autoimmun. 2020 Mar;108:102417. Innate Inflammation Drives NK Cell Activation to Impair Treg Activity. Dean JW et al. University of Florida, Gainesville, FL, USA. Link: Abstract
Semin Immunopathol. 2019 Jan;41(1):59-68. Off-the-shelf Cell Therapy With Induced Pluripotent Stem Cell-Derived Natural Killer Cells. Saetersmoen ML et al. University of Oslo, Oslo, Norway
Results / Comments: In this review, the group outlines a roadmap for development of off-the-shelf cell therapy based on natural killer (NK) cells derived from induced pluripotent stem cells (iPSCs). Link: Abstract .You can also click here to get full-page newspaper articles.

● Antisense ATL1102 final phase II DMD results meet primary endpoint and exceed expectations on secondary endpoints
Antisense Therapeutics announced on May 21s that the Phase II clinical trial of ANP’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) has met its primary endpoint confirming the safety and tolerability of ATL1102 for advancement into a potentially pivotal Phase IIb clinical trial. Importantly, the final trial results have also confirmed the drug’s positive effects on the secondary trial endpoints that assessed the drug’s activity and efficacy including measuring the effects on immune cell numbers in the blood and measuring the participants’ functional capacity as evaluated via Performance of Upper Limb Test (PUL2.0), grip and pinch strength and distal mobility (using the MyoSet, MyoGrip, MyoPinch and MoviPlate tools, respectively). Additionally, the Company is very pleased to report that MRI assessment of the upper limb muscles of the patients with DMD has also shown the drug’s apparent beneficial effects in stabilising the fat fraction percentage within the muscles of the forearm (increase in fat levels is another key marker of disease progression in non-ambulant DMD boys). The data shows a stabilisation in the percentage of fat in the forearm muscles and an increase/maintenance of functional muscle mass, which is both outstanding and unexpected for a drug treating the inflammation (and not the muscle dystrophin loss). For further informations

Related Informations / Publications
MAR 2020: ATL1102 Phase II DMD trial data to be presented at Duchenne ACTT Now Conference.  Link: Press Release
DEC 2019: Positive results reaffirm plans to advance ATL1102 for DMD to Phase IIb trial
Results / Comments: ATL1102 appears to be generally safe and well tolerated in non-ambulant boys with DMD. No Serious Adverse Events have been reported and there have been no safety concerns expressed by the Data Safety Monitoring Board. Link: Press Release
JUL 2019: Phase II clinical trial in DMD on track for dosing completion.
Results / Comments: The open label six-month dosing trial of ATL1102 in nine non-ambulant patients with DMD aged between 10 and 18 years is being conducted at the neuromuscular centre of the Royal Children’s Hospital in Melbourne which operates the largest clinic in the southern hemisphere treating children with DMD. Link: Press Release
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● Gilead Sciences and Arcus Biosciences establish 10-year partnership to co-develop and co-commercialize next-generation cancer immunotherapies
Gilead Sciences and Arcus Biosciences, an oncology-focused biopharmaceutical company working to create best-in-class cancer therapeutics, announced on May 27th that the companies have entered into a 10-year partnership to co-develop and co-commercialize current and future therapeutic product candidates in Arcus’s pipeline. The agreement will also provide ongoing funding to support Arcus’s research and development programs. Arcus is building an extensive and diverse portfolio of investigational products that target important mechanisms involved in tumor evasion of the immune system, as well as developing drug candidates that target cell-intrinsic pathways important for cancer growth and metastasis. In addition to small molecule products, Arcus is also advancing antibody products that target immune checkpoint receptors, including PD-1 and TIGIT. Arcus currently has a clinical-stage pipeline of four immuno-oncology programs, as well as an active oncology discovery pipeline with six preclinical compounds that target critical biological pathways. A core component of Arcus’s strategy is the development of intra-portfolio combinations that include small-molecule and antibody product candidates. Arcus has 10 ongoing clinical studies of molecules in its portfolio, including a randomized Phase 2 study in first-line non-small cell lung cancer evaluating combinations of three Arcus product candidates: AB154, an investigational anti-TIGIT monoclonal antibody; AB928, an investigational A2aR/A2bR antagonist; and zimberelimab (AB122), an investigational anti-PD-1 monoclonal antibody. For further information

Related Informations / Publications
MAY 2020: Arcus Biosciences Announces Commencement of Underwritten Public Offering of Common Stock. Link: Press Release
Cell. 2020 Apr 30;181(3):728-744.e21. Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies. Roth TL et al. University of California, San Francisco, San Francisco, CA, USA.
Results / Comments: Pooled knockin screening enables parallelized re-writing of endogenous genetic sequences to accelerate discovery of knockin programs for cell therapies. Link: Abstract
DEC 2019: Arcus Biosciences, in Collaboration with Genentech, Announces Two Randomized Clinical Studies to Advance AB928, a Dual Adenosine Receptor Antagonist, into Novel Combinations for Colorectal and Pancreatic Cancers. Link: Abstract
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● Merck to acquire Themis
Merck&Co and Themis, a company focused on vaccines and immune-modulation therapies for infectious diseases and cancer, announced on May 26th that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire privately-held Themis. Themis has a broad pipeline of vaccine candidates and immune-modulatory therapies developed using its innovative measles virus vector platform based on a vector originally developed by scientists at the Institut Pasteur, a world-leading European vaccine research institute, and licensed exclusively to Themis for select viral indications. In March, Themis joined a consortium together with the Institut Pasteur and The Center for Vaccine Research at the University of Pittsburgh, supported by funding from the Coalition for Epidemic Preparedness Innovations (CEPI), to develop a vaccine candidate targeting SARS-CoV-2 for the prevention of COVID-19. For further information

Related Informations / Publications
MAY 2020: Themis collaborates with ABL Europe to manufacture its SARS-CoV-2 Vaccine Candidate in France. Link: Press Release
MAR 2020: Themis and Institut Pasteur Join Coronavirus Vaccine Hunt. Link:
AUG 2019: Themis Bioscience Announces Exclusive License and Research Collaboration Agreement with MSD to Develop Vaccine Candidates. Link: Press Release
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● Alnylam announces US FDA has granted priority review of the Lumasiran NDA for the treatment of primary hyperoxaluria type I
Alnylam Pharmaceuticals, a leading RNAi therapeutics company, announced on May 26th that the FDA has accepted the Company’s New Drug Application (NDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). The FDA also granted Priority Review for the NDA, a designation for medicines that have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease, with the goal of FDA taking action within six months compared to 10 months under standard review. In addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA). Lumasiran was previously granted an accelerated assessment by the EMA, which is awarded to medicines deemed to be of major public health interest and therapeutic innovation, and is designed to bring new treatments to patients more quickly. Accelerated assessment potentially reduces the Agency’s evaluation time from 210 to 150 days. For further information

Related Informations / Publications
MAY 2020: Alnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTRO® (patisiran) at the European Academy of Neurology Virtual Congress 2020.  Link: Press Release
-APR 2020: Blackstone and Alnylam Enter Into $2 Billion Strategic Financing Collaboration to Accelerate the Advancement of RNAi Therapeutics. Link: Press Release
Elife. 2020 Mar 24;9:e54363. Characterising a Healthy Adult With a Rare HAO1 Knockout to Support a Therapeutic Strategy for Primary Hyperoxaluria. McGregor et al. Alnylam Pharmaceuticals, Cambridge, United States. Link: AbstractFull Text
-MAR 2020: Alnylam Announces Approval of GIVLAARI® (givosiran) in the European Union for the Treatment of Acute Hepatic Porphyria (AHP) in Adults and Adolescents. Link: Press Release
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● Orgenesis announces collaboration with Hospital Infantil Universitario Niño Jesús, Madrid
Orgenesis, a pioneering global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs) while lowering costs, announced on May 22nd a research and development collaboration with Hospital Infantil Universitario Niño Jesús, Madrid, Spain, through its Biomedical Research Foundation, FIB, to establish a point-of-care center leveraging Orgenesis’ cell and gene therapy focused POCare Platform. Under the collaboration agreement, FIB Hospital Infantil Universitario Niño Jesús will utilize Orgenesis’ CGT Biotech Platform to develop, optimize and manufacture cell and gene therapies. Orgenesis’ POCare Technologies enable hospitals to develop and supply cell and gene therapies, including Orgenesis’ proprietary POCare Therapies, by implementing Orgenesis’ proprietary know-how combined with automated, closed technology to process select cell therapies at each point-of-care site for the treatment of patients. The first collaboration under the agreement between FIB Hospital Infantil Universitario Niño Jesús and Orgenesis involves the clinical development and validation of T-cell and dendritic cell-based therapies. Orgenesis’ CGT Biotech Platform, which combines processing and therapeutic technologies, is designed to allow for the efficient production of high quality, affordable cell and gene-based products. Upon successful completion of the first collaboration, Orgenesis and FIB Hospital Infantil Universitario Niño Jesús then plan to pursue the development of the Hospital’s oncolytic based cell therapy technology, Celyvir. This proprietary program represents a new strategy for the treatment of solid metastatic tumors based on oncolytic virotherapy administered by mesenchymal cells obtained from bone marrow. For further informations, see Orgenesis. See also Vered Caplan, CEO of Orgenesis, interview with the Editor-In-Chief at Cell & Gene, about the sale of its CDMO subsidiary and the role of its new Cell & Gene Therapy (CGT) Biotech Platform in driving down cell therapy cost.

Related Informations / Publications
APR 2020: Orgenesis Announces Agreement to Acquire Assets of Tamir Biotechnology, Inc. Including Broad Spectrum Antiviral Platform. Link: Press Release
APR 2020: Orgenesis Announces Joint Venture Agreement with RevaTis to Produce Muscle-Derived Mesenchymal Stem Cells (mdMSC) as a Source of Exosomes and Other Cellular Products for the Development of Related Therapies. Link: Press Release
MAR 2020: Orgenesis and ExcellaBio Announce Breakthrough Manufacturing Process for Bioxomes™, Proprietary Synthetic Exosomes/Extracellular Vesicles (EV). Link: Press Release
FEB 2020: Orgenesis Announces Collaboration with The John Hopkins University for the Development and Processing of Cell and Gene Based Clinical Therapeutics. Link: Press Release
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● Novasep and Lysogene announce their new collaboration for the development and production of GM1 gangliosidosis gene therapy product
Novasep, a leading supplier of services and technologies for the life sciences industry, and Lysogene, a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, announced on May 25th the signature of an agreement for the development and manufacturing of LYS-GM101, an AAVrh10-based gene therapy drug candidate for the treatment of GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder. With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene’s lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3. For further information

Related Informations / Publications
MAR 2020: Lysogene completes €7.7 million capital increase. Link: Press Release
-FEB 2020: Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA. Link: Press Release
-DEC 2019: Novasep launches oXYgene™, a fully integrated offer for boosting viral vector production. Link: Press Release
APR 2019: NOVASEP and THERAVECTYS enter into a new license agreement on DNA Flap Technology for GMP Manufacturing of lentiviral vectors
Results / Comments: This agreement supports the growing demand for lentivector-based gene therapies, immunotherapies and vaccines. Link: Press Release
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