Trend Chart on Innovative Bioindustries April 12th
Trend Chart On....

Trend Chart on Innovative Bioindustries April 12th

Trend Chart on Innovative Bioindustries April 12th


Sirnaomics releases positive interim data from phase II Trial of siRNA candidate studies
Sirnaomics, a US-China RNAi therapeutics company, reported on April 9th positive interim results from a Phase II trial of its RNA treatment for non-melanoma skin cancer. STP705 is a siRNA (small interfering RNA) candidate that produced a complete histologic clearance of squamous cell carcinoma in situ (isSCC) in ten of fifteen patients. The candidate uses a polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-β1 and COX-2 gene expression. The results came from the first three cohorts of a US Phase II dose-escalation trial. Sirnaomics is headquartered in Maryland with subsidiaries in Suzhou and Guangzhou. For further information

Related Informations / Publications
Brief Bioinform. 2020 Apr 6. The mechanistic, diagnostic and therapeutic novel nucleic acids for hepatocellular carcinoma emerging in past score years. Zhang S et al. College of Pharmaceutical Sciences in Zhejiang University, China (Abstract)
Results/Comments: This review takes a detailed look at the advances of novel nucleic acids from potential of biomarkers and elaboration of mechanism to early clinical application in past 20 years.
FEB 2020: Sirnaomics’ siRNA Therapeutic Candidate, STP705, Granted Orphan Drug Designation By US FDA for Treatment of Hepatocellular Carcinoma.  Press Release
JAN 2020: Sirnaomics and Junshi Biosciences Entered Strategic Collaboration to Conduct Clinical Oncology Studies Combining Tuoyi and RNAi in China and US. Press Release
Sci Rep. 2018 Aug 28;8(1):12954. Evaluation of modified Interferon alpha mRNA constructs for the treatment of non-melanoma skin cancer. Hochmann S et al. Paracelsus Medical University, Salzburg, Austria. (Abstract)

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T cell-engaging therapies — BiTEs and beyond
Immuno-oncology approaches have entered clinical practice, with tremendous progress particularly in the field of T cell-engaging therapies over the past decade. In a recent review, German researchers provide an overview of the current status of bispecific T cell engager (BiTE, therapy, considering the unprecedented new indication for such therapy in combating minimal (or measurable) residual disease in patients with acute lymphoblastic leukaemia, and the development of novel approaches based on this concept. Key aspects that we discuss include the current clinical data, challenges relating to treatment administration and patient monitoring, toxicities and resistance to treatment, and novel strategies to overcome these hurdles as well as to broaden the indications for BiTE therapy, particularly to common solid cancers. Elucidation of mechanisms of resistance and immune escape and new technologies used in drug development pave the way for new and more-effective therapies and rational combinatorial approaches. In particular, they highlight novel therapeutic agents, such as bifunctional checkpoint-inhibitory T cell engagers (CiTEs), simultaneous multiple interaction T cell engagers (SMITEs), trispecific killer engagers (TriKEs) and BiTE-expressing chimeric antigen receptor (CAR) T cells (CART.BiTE cells), designed to integrate various immune functions into one molecule or a single cellular vector and thereby enhance efficacy without compromising safety. They also discuss the targeting of intracellular tumour-associated epitopes using bispecific constructs with T cell receptor (TCR)-derived, rather than an antibody-based, antigen-recognition domains, termed immune-mobilizing monoclonal TCRs against cancer (ImmTACs), which might broaden the armamentarium of T cell-engaging therapies. The review appared in April 02nd online issue of Nat Rev Clin Oncol

Related Informations / Publications
Curr Med Sci. 2020 Feb;40(1):28-34. Selection for Anti-transferrin Receptor Bispecific T-cell Engager in Different Molecular Formats. Fu MP et al. Huazhong University of Science and Technology, Wuhan, 430030, China (Abstract)
Oncologist. 2019 Nov 14. pii: theoncologist.2019-0559. Blinatumomab for Acute Lymphoblastic Leukemia: The First Bispecific T-Cell Engager Antibody to Be Approved by the EMA for Minimal Residual Disease. Ali S et al. European Medicines Agency, Amsterdam, The Netherlands (Abstract)
Results/Comments: New recommendations and change of practice for treatment of this patient group are detailed
Antibodies (Basel). 2019 Aug 2;8(3). Design and Production of Bispecific Antibodies. Wang J et al. Johns Hopkins University, Baltimore, MD 21218, USA (AbstractFull Text)
Results/Comments: In this review, researchers focus on these two major bispecific types and describe recent advances in the design, production, and quality of these molecules, which will enable this important class of biologics to reach their therapeutic potential.

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Seneca completes stem cell manufacturing plant in Suzhou
Seneca Bio, a Maryland biopharma, said on April 3rd that it has completed its new Suzhou cell manufacturing facility that will manufacture the company’s stem cell therapy for ischemic stroke. NSI-566 is currently being tested in a non-GCP compliant Phase II trial at the BaYi Brain Hospital in Beijing. A spinal-cord derived neural stem cell line. NSI-566 is being investigated as a treatment for stroke, Amyotrophic Lateral Sclerosis (ALS), and chronic spinal cord injury. Seneca was previously known as Neuralstem. For further information, see ChinaBio Today

Related Informations / Publications
NOV 2019: Seneca Biopharma and Jiangsu QYuns Therapeutics Announce Development Plan for Potential Joint Pipeline at 7th China Renaissance Healthcare and Life Science Leadership Summit (Press Release)
Stem Cells Transl Med. 2019 Oct;8(10):999-1007. Stable Intracerebral Transplantation of Neural Stem Cells for the Treatment of Paralysis Due to Ischemic Stroke. Zhang G et al. Affiliated BaYi Brain Hospital, Army General Hospital of PLA, Beijing, China (AbstractFull Text)
Results/Comments: Longitudinal MRI studies showed evidence indicating cavity-filling by new neural tissue formation in all nine patients.
SEP 2019: Neuralstem Announces Last Subject Enrolled in Phase 2 Clinical Trial Evaluating Human Neural Stem Cells in Chronic Stroke Patients (Press Release)
Oper Neurosurg (Hagerstown). 2019 Aug 14. First Human Trial of Stem Cell Transplantation in Complex Arrays for Stroke Patients Using the Intracerebral Microinjection Instrument. Zhang G et al. Affiliated BaYi Brain Hospital, Army General Hospital of PLA, Beijing, China (Abstract)


● Voyager Therapeutics provides update on the RESTORE-1 Parkinson’s disease trial
Voyager Therapeutics, a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, provided on April 3rd an update on the RESTORE-1 clinical trial of VY-AADC (NBIb-1817) for Parkinson’s disease. Voyager and its collaborator Neurocrine Biosciences have temporarily paused screening of new patients in the RESTORE-1 trial to allow clinical trial sites to assess the impact of the COVID-19 pandemic on both the implementation of previously disclosed protocol amendments and on the safety of study participants. The companies plan to resume patient screening and enrollment as soon as the assessment is completed and Neurocrine, as the study sponsor, and Voyager, in consultation with clinical sites, determine that screening can safely and effectively resume. Voyager and Neurocrine Biosciences continue preparations for the initiation of the RESTORE-2 registrational study in Parkinson’s disease planned for the second half of 2020. For further information.

Related Informations / Publications
Mov Disord. 2020 Mar 9. doi: 10.1002/mds.27993. Aromatic L-Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson’s Disease. Nutt JG et al. Oregon Health & Science University, Portland, Oregon, USA (Abstract)
Parkinsonism Relat Disord. 2019 Sep;66:16-24. Human gene therapy approaches for the treatment of Parkinson’s disease: An overview of current and completed clinical trials. Hitti FL et al. University of Pennsylvania, 800 Spruce St, Philadelphia, PA 19107, USA (Abstract)
MAY 2019: Neurocrine Biosciences and Voyager Therapeutics Announce Phase I Results for VY-AADC in Patients with Parkinson’s Disease at the American Academy of Neurology Annual Meeting
Results/Comments: Treatment with VY-AADC improved good ON time (ON time without troublesome dyskinesia) by 1.7 hours from baseline and reduced OFF time by 2.2 hours at 12 months from baseline in patients with Parkinson’s disease (Press Release)
JAN 2019: Neurocrine Biosciences and Voyager Therapeutics Form Strategic Development and Commercialization Collaboration for Parkinson’s Disease and Friedreich’s Ataxia (Press Release)



● Dynacure secures $55M as their Ionis-developed drug moves into clinic
A month after dosing their first ever patient with their Ionis-licensed tech, Dynacure has announced on April 2nd a new round of funding to advance the trial. The French biotech announced €50 million ($55 million) in Series C funding, led by Perceptive Advisors. Although listed as a Series C, it is the second tranche of cash the company has ever announced, after their initial $55 million raise in 2018. The cash will float Dynacure through its Phase I/II trial in a class of neuromuscular diseases known as myotubular and centronuclear myopathies. The lead compound they developed uses antisense to target Dynamin-2, a gene implicated in some forms of the disease. As cells send RNA messages to build the protein, the antisense binds to the RNA, blocking designated base pairs and either changing the protein that is ultimately produced or significantly lowering the amount of protein produced. A Phase I/II trial began last month on patients over 16 with one of two forms of the genetic disorder, either X-linked or autosomal dominant. A third — known as autosomal recessive — is mediated by a different gene.
For further information, see Endpoint News

Related Informations / Publications
MAR 2020: Dynacure Announces First Patient Dosed in Phase 1 / 2 ‘UNITE-CNM’ Study of DYN101 for the Treatment of Myotubular and Centronuclear Myopathies (CNM) (Press Release)
Hum Mol Genet. 2019 Dec 15;28(24):4067-4077. Different in vivo impacts of dynamin 2 mutations implicated in Charcot-Marie-Tooth neuropathy or centronuclear myopathy. Massana Muñoz X et al. Institut de Génétique et de Biologie Moléculaire et Cellulaire, Illkirch, France (Abstract)
OCT 2019: Dynacure Presents New Preclinical Results on Muscle Targeting with Antisense Oligonucleotides at the 24th Annual International Congress of the World Muscle Society (Press Release)
Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. Reducing dynamin 2 (DNM2) rescues DNM2-related dominant centronuclear myopathy. Buono S et al. Institut de Génétique et de Biologie Moléculaire et Cellulaire, 67404 Illkirch, France ( AbstractFull Text)
Results/Comments: DNM2 knockdown via two different strategies can efficiently correct the myopathy due to DNM2 mutations, and it provides a common therapeutic strategy for several forms of centronuclear myopathy.


Orgenesis Announces Joint Venture agreement with RevaTis to produce muscle-derived mesenchymal stem cells
Orgenesis, a vertically integrated biotech company committed to lowering costs, accelerating commercialization, and transforming the delivery of cell and gene therapies (CGT) through its Point-of-Care (POCare) Platform, announced on April 8th a joint venture agreement with RevaTis. The goal of the JV is to advance the development of autologous therapies, including in-licensed therapies through Orgenesis’s partners, utilizing and banking muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and other cellular products. RevaTis has developed a patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy and produce mdMSCs utilizing a turnkey isolator system. The goal of the JV is to build upon RevaTis’ initial success in animals to develop therapies and advance human trials by leveraging Orgenesis’s technical, clinical and regulatory expertise, as well as Orgenesis’s POCare Technologies. These technologies include automated/closed-systems, 3D printing, and bioreactor technologies. The JV will be dedicated to developing RevaTis’ technology in humans. For further information

Related Informations / Publications
MAR 2020: Orgenesis and ExcellaBio Announce Breakthrough Manufacturing Process for Bioxomes™, Proprietary Synthetic Exosomes/Extracellular Vesicles (Press Release)
FEB 2020: Orgenesis Announces Collaboration with The John Hopkins University for the Development and Processing of Cell and Gene Based Clinical Therapeutics (Press Release)
FEB 2020: Orgenesis Completes Sale of Masthercell Subsidiary; Receives Approximately $127 Million in Net Proceeds (Press Release)

Fate Therapeutics announces worldwide collaboration with Janssen for novel iPSC-derived Cell-based cancer immunotherapies
Fate Therapeutics, a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced on April 02nd a global collaboration and option agreement with Janssen Biotech. Under the multi-year collaboration agreement, Janssen will contribute proprietary antigen binding domains for up to four tumor-associated antigen targets. The company will apply its iPSC product platform to research and preclinically develop new iPSC-derived chimeric antigen receptor (CAR) NK and CAR T-cell product candidates. The company will receive $50 million in cash and $50 million from the purchase by Johnson & Johnson Innovation – JJDC, Inc. of newly issued shares of the Company’s common stock at a price per share of $31.00. Janssen will also reimburse the company for all activities conducted under the collaboration. The company will advance candidates under the collaboration to the filing of an Investigational New Drug (IND) application, after which Janssen will have the right to exercise its option for an exclusive license for the development and commercialization of collaboration candidates targeting the tumor-associated antigens. The company will be primarily responsible for the manufacture of collaboration candidates, the cost of which will be paid for by Janssen. The company is eligible to receive payments of up to $1.8 billion upon the achievement of development and regulatory milestones and up to $1.2 billion upon the achievement of commercial milestones, plus double-digit royalties on worldwide commercial sales of products targeting the antigens. In addition, the company has the right to elect to co-commercialize each collaboration candidate in the U.S. and share equally in profits and losses in the U.S., subject to its payment of certain clinical development costs and adjustments in milestone and royalty payments. For further information

Related Informations / Publications
Bone Marrow Transplant. 2020 Mar 13. doi: 10.1038/s41409-020-0858-9. Presence of donor-encoded centromeric KIR B content increases the risk of infectious mortality in recipients of myeloablative, T-cell deplete, HLA-matched HCT to treat AML. Bultitude WP et al. Anthony Nolan Research Institute, Royal Free Hospital, London, UK (Abstract)
JAN 2020: Fate Therapeutics Announces Expansion of FT516 Clinical Investigation and Publication of Preclinical Data in the Journal Blood
Results/Comments: FT516 IND Application Cleared by FDA for Advanced Solid Tumors in Combination with PDL1-, EGFR- and HER2-targeting Therapeutic Antibodies (Press Release)
DEC 2019: Fate Therapeutics Announces New Preclinical Data for FT596 Off-the-Shelf, iPSC-derived CAR NK Cell Cancer Immunotherapy (Press Release)
Results /Comments: FT596 as a Monotherapy Demonstrates Comparable Anti-tumor Activity to CAR19 T Cells In Vivo in Humanized Mouse Model of Lymphoma

Alnylam and Dicerna form RNAi Therapeutics collaboration on Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Alnylam Pharmaceuticals and Dicerna Pharmaceuticals, both leaders in the field of ribonucleic acid interference (RNAi) therapeutics, announced on April 6th the formation of a development and commercialization collaboration on investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency associated liver disease (alpha-1 liver disease). In addition, the companies have completed a cross-license of their respective intellectual property for Alnylam’s lumasiran and Dicerna’s nedosiran investigational programs for the treatment of primary hyperoxaluria. These agreements will enhance and accelerate Alnylam’s and Dicerna’s ability to bring these orphan product candidates to market. Under the development and commercialization agreement, Alnylam’s ALN-AAT02 and Dicerna’s DCR-A1AT, investigational RNAi therapeutics, each in Phase 1/2 development, will be explored for the treatment of alpha-1 liver disease. Under the agreement, Dicerna assumes responsibility for both ALN-AAT02 and DCR-A1AT at its cost, and may progress one or both of these investigational medicines through clinical development. Dicerna will select which product candidate(s) to advance in development for the treatment of patients with alpha-1 liver disease. Alnylam also announed on April 6th that it has completed rolling submission of NDA to the US FDA and submitted AMM to the EMA for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1. For further information

Related Informations / Publication
Elife. 2020 Mar 24;9. pii: e54363. Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria. McGregor TL et al. Alnylam Pharmaceuticals, Cambridge, USA (AbstractFull Text)
MAR 2020: Dicerna Provides Initial Observations From PHYOX™3 Trial of Nedosiran for Treatment of Primary Hyperoxaluria and an Update on Data Presentation Plan (Press Release)
MAR 2020: Alnylam Announces Approval of GIVLAARI® (givosiran) in the European Union for the Treatment of Acute Hepatic Porphyria (AHP) in Adults and Adolescents (Press Release)
FEB 2020: Alnylam Completes Enrollment of HELIOS-A Phase 3 Study of Vutrisiran for the Treatment of hATTR Amyloidosis with Polyneuropathy (Press Release)
Expert Opin Drug Deliv. 2018 Jun;15(6):629-640. Recent preclinical and clinical advances in oligonucleotide conjugates. Craig K et al. School of Pharmacy, Northeastern University , Boston , MA, USA (Abstract)
Results/Comments: Synthesis of these approaches has the potential to bring the next breakthrough in oligonucleotide therapeutics.


Polyplus-transfection launches FectoVIR-AAV transfection reagent for CGT development viral vector bottleneck
Polyplus-transfection, a leading biotechnology company that supports the gene and cell therapy market by supplying innovative transfection solutions, announced on April 06th it will make available to the market a novel transfection reagent FectoVIR(R)-AAV in May 2020. The reagent has been specifically developed to improve AAV (adeno-associated virus) production in suspension cell culture system for large-scale manufacturing. AAVs are predominantly used in gene therapy development. To date there has been a significant and well-identified bottleneck in manufacturing sufficient amounts of AAV viral vectors to treat patients. This will continue to increase as more therapies are commercialized, and reach late stage clinical trials. Viral manufacturers, in order to increase capabilities, are looking for solutions to adapt key steps of production for large-scale manufacturing. Transient transfection is a critical step that is indispensable for a reliable industrial manufacturing process. Polyplus-transfection has developed the FectoVIR-AAV transfection reagent to achieve large-scale transient transfection resulting in up to a 10-fold increase in AAV functional viral titer yields in suspension cell systems. Polyplus-transfection developed the FectoVIR-AAV transfection reagent in partnership with a range of viral manufacturers internationally. Rather than follow the industry standard model of developing a reagent for small-scale manufacturing and adapting for large-scale production, Polyplus-transfection has developed the entirely new reagent with large-scale transient transfection in mind, and then validated its use all the way through the R&D process. For further information

Related Informations / Publications
Oncotarget. 2018 Nov 13;9(89):35891-35906. Oncolytic properties of non-vaccinia poxviruses. Ricordel M et al. Transgene SA, Illkirch-Graffenstaden 67405, France (AbstractFull Text)Results/Comments: The results demonstrated significant antitumoral activity of this new modified Raccoonpox virus armed with FCU1 and this virus could be considered to be included into the growing armamentarium of oncolytic virotherapy for cancer
Cancers (Basel). 2018 Jul 10;10(7). Vaccinia Virus Shuffling: deVV5, a Novel Chimeric Poxvirus with Improved Oncolytic Potency. Ricordel M et al. Transgene SA, Illkirch-Graffenstaden 67405, France (AbstractFull Text)
Oncotarget. 2018 Nov 13;9(89):35891-35906. Oncolytic properties of non-vaccinia poxviruses. Ricordel M et al. Transgene SA, Illkirch-Graffenstaden 67405, France
Results/Comments: The results demonstrated significant antitumoral activity of this new modified Raccoonpox virus armed with FCU1 and this virus could be considered to be included into the growing armamentarium of oncolytic virotherapy for cancer (AbstractFull Text)

Cobra Biologics successfully completes key milestone in CombiGene’s epilepsy project CG01
Cobra Biologics, an international CDMO for biologics and pharmaceuticals, and CombiGene, a leading Nordic gene therapy company, announced on April 2nd the successful production and delivery, by Cobra, of the first DNA plasmid required to produce CombiGene’s gene therapy drug candidate CG01, developed for the treatment of drug-resistant focal epilepsy.The production of compounds for a novel gene therapy depends on cultivated and modified cells, and is therefore a more complex process than for traditional drug candidates. For the vital compounds of CG01, Cobra Biologics has finalised production and delivery of the first of three key plasmids, enabling the downstream processes to be initiated. In addition to these plasmids, a specific type of cell, which Cobra is also producing, acting as the ‘factory’ to produce the CG01 compound is necessary. After the production of the plasmids for CG01, and the successful transfer of the production process from the Cell and Gene Therapy Catapult to Cobra, an engineering run will be initiated, whereby the entire production process will be tested. Material from this engineering run will be used in the toxicity and biodistribution tests that are planned. For further information

Related Informations / Publications
MAR 2020 : CombiGene has concluded a successful learning and memory study in the CG01 epilepsy project (Press Release)
JAN 2020 : Cognate BioServices closes Series B and completes acquisition of Cobra Biologics
Results/Comments: Cobra Biologics has cGMP facilities in the UK and Sweden.  Cognate operates an 80,000+ square foot manufacturing facility located in Memphis, TN, and a dedicated development facility in Baltimore (Press Release)
Mol Ther Methods Clin Dev. 2019 Sep 18;15:179-193. Disease Modification by Combinatorial Single Vector Gene Therapy: A Preclinical Translational Study in Epilepsy. Melin et al. Lund University Hospital, Sölvegatan 17, 221 84 Lund, Sweden (AbstractFull Text)