spacer
# http://biopharmanalyses.fr ›
 spacer
 spacer
 spacer
Trend Chart oN
INNOVATIVE BIOindustries
Dearl all,
 Please, find below the latest edition of
our TrendChart on Innovative Bioindustries.
You will find here 10 selected items not older than 10 days.
These news are placed in context with the latest relevant informations.
 In each issue, we also highlight a feature story that presents
 an accurate and meaningful information in the biotech area.
Feel free to contact us and we will happy to build with you the tools you need to strategically address opportunities available in your business area
(competitive landscape, follow-up of selected companies, follow-up of product portfolio in a defined therapeutic area or in defined disorders….) 
We hope you’ll enjoy reading Trend Chart on Innovative Bioindustries
Anne-Lise Berthier 
General Manager
BioPharmAnalyses
 spacer
alb@biopharmanalyses.fr
Phone: 33 (0) 686 683 220 
 spacer
Olivier Revelant
General Manager
Octopusyx BioConsulting
 spacer
olivier@octopusyx.fr
Phone: 33 (0) 668 071 907
 spacer
 spacer
contents
October 2019, 12th

FEATURE STORY
●  Bluebird bio and Novo Nordisk enter into research agreement to develop in vivo genome editing candidates 
CLINICAL TRIALS - DATAS
● Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit
● ProQR announces positive top-line results from the phase 1/2 study of Sepofarsen in LCA10 patients
● Ionis' Factor XI anti-thrombotic medicine advances with Bayer following positive clinical results
M&A - AGREEMENTS
● Akcea and Pfizer announce licensing agreement for investigative antisense therapy AKCEA-ANGPTL3-L(Rx)
● Editas Medicine and MaxCyte announce clinical and commercial license agreement for engineered cell medicines
● PTC Therapeutics establishes strategic collaboration with Aldevron to support GMP plasmid manufacturing
● Lonza agrees to manufacture Prevail Therapeutics’ novel AAV-based gene therapies for patients with neurodegenerative diseases
● Cellectis and Lonza enter cGMP manufacturing service agreement for Cellectis’ allogeneic UCART product candidates
● Alexion and Stealth announce agreement for option to co-develop and commercialize late-stage therapy for mitochondrial diseases
● TrakCel announces strategic partnership with WuXi AppTec Advanced Therapies for optimization of supply chain for cell and gene therapy treatments

"Landscape in.... Gene Therapy Companies"
Now Published
We are proud to announce the launch of the enriched edition of our
"Landscape... in Gene Therapy Companies"
(Updated SEPTEMBER 2019)
This report provides you with an in-depth analysis of
more than 230 companies involved in this expanding field. 
In this 800-pages report, a must-have read one, you will find included:
More than 230 closely scrutinized companies
More than 600 products identified including more than
200 products at the clinical stage 
More than 300 VC or investors mentioned 
More than 250 pathologies referred.
A Product Breakown by Development Phase

A Detailed Overview of Companies Involved in …
AAV-based Gene Therapy
LV-based Gene Therapy
RNA-based Derived Drugs
(RNAi, mRNA Therapeutics...)
Gene Editing Therapy
 CDMO (Contract, Development and Manufacturing Organisation)
Miscellaneous (Including Hubs and Non Viral Gene Transfer)*
* CAR-T strategies will be dealt separately in the updated edition of our briefing
on CAR-T clinical trials. The 2019 edition will be published next November.  
Each Technical Datasheet includes for Every Company: spacer
General Informations (location, website, FTE, management with e-mail contacts) - Core Business in Gene Therapy 
Financial Data (stock informations, market cap, financial highlights, recent fundings including main investors)  
Pipeline (discovery, pre-clinical, phase I / II / III, marketed products)
Orphan Drug Designations 
Clinical Trials 
Latest Developments in Gene Therapy
Latest Publications 

«LANDSCAPE IN GENE THERAPY COMPANIES» 
 €3490 (UPDATED SEP 2019)
Please click here to get the executive content and some technical datasheet
you will be able to find in the final global report

 spacer
 spacer
FEATURE STORY
Bluebird bio and Novo Nordisk enter into research agreement to develop in vivo genome editing candidates
Bluebird bio and Novo Nordisk announced on October 09th that they have entered into a research collaboration to jointly develop next-generation in vivo genome diting treatments for genetic diseases, including haemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate with the ambition of offering people with haemophilia A a lifetime free of factor replacement therapy. The research collaboration will utilise bluebird bio’s proprietary mRNA-based megaTAL™ technology that has the potential to provide a highly specific and efficient way to silence, edit or insert genetic components. Aligned with Novo Nordisk’s haemophilia portfolio, the research collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
For further information 
Related Informations/Publications
-Appl Clin Genet. 2019 Sep 23;12:167-180. Gene Therapy For Beta-Thalassemia: Updated Perspectives. Karponi G et al. Aristotle University of Thessaloniki, Thessaloniki, Greece . Link: Abstract – Full Text
-JUN 2019: bluebird bio Presents Long-Term Efficacy and Safety Data from Clinical Studies of LentiGlobin® Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) at 24th European Hematology Association (EHA) Congress
Results/Comments: The results reported for the completed Phase 1/2 Northstar (HGB-204) study reflect data as of December 13, 2018; of the 18 patients in the study, 10 patients do not have a β0/β0 genotype and eight have a β0/β0 genotype. All 18 patients have completed the two-year study and enrolled in the long-term follow-up study, LTF-303. Link: Press Release
-JUN 2019: Bluebird prices gene therapy at 1.58 million euros over 5 years Results/Comments: Bluebird bio Inc set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe to treat a rare genetic blood disorder. Link: Reuters
-N Engl J Med. 2018 Apr 19;378(16):1479-1493. Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia. Thompson AA et al. Northwestern University Feinberg School of Medicine, Chicago, USA
Results/Comments: Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long-term red-cell transfusions in 22 patients with severe β-thalassemia without serious adverse events related to the drug product. Link: Abstract  - Full Text
-MAY 2019: €60M Investment to Boost Oxford Biomedica’s Gene and Cell Therapies Results/Comments: The Danish investor Novo Holdings is handing Oxford Biomedica up to €60.6M (£53.5M) to develop gene and cell therapies treating cancer and genetic blindness. Link: Press Release  
 spacer
 spacer
 spacer
CLINICAL TRIALS - DATAS
Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit
PTC Therapeutics announced on October 02nd data demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA) at the 24th International Annual Congress of the World Muscle Society. Presentations include data from the FIREFISH, SUNFISH, and JEWELFISH clinical trials. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche. Data from 45 patients in the JEWELFISH trial demonstrated a sustained, greater than two-fold increase in median SMN protein versus baseline over 12 months of treatment. Patients in the JEWELFISH study have previously been treated with nusinersen or other therapies. In August, the first patient was enrolled in the RAINBOWFISH trial, an open-label, single-arm, international, multi-center clinical study to investigate the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam in infants with genetically diagnosed SMA who are not yet presenting symptoms. Risdiplam has been well tolerated at all dose levels and in all clinical studies. To date, there have been no drug-related safety findings leading to withdrawal from any study.
For further information  
Related Informations / Publications
-SEP 2019: Novartis blames former AveXis executives for Zolgensma data manipulation Link: Reuters
-Pediatr Neurol. 2019 Jun 13. pii: S0887-8994(18)31163-9. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Al-Zaidy SA et al. Nationwide Children's Hospital, Columbus, Ohio, USA Results/Comments: This review discusses the biological rationale underlying gene replacement therapy for spinal muscular atrophy, describes the onasemnogene abeparvovec clinical trial experience, and provides expert recommendations as a reference for the real-world use of onasemnogene abeparvovec in clinical practice
Link: Abstract - Full Text
 -Prog Med Chem. 2019;58:119-156. Rewriting the (tran)script: Application to spinal muscular atrophy. Ratni H et al. Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
Results/Comments: This review discusses the vital but feasible requirement for such drugs to deliver selectivity, and critical safety aspects are highlighted
Link: Abstract
-MAY 2019: Roche risdiplam data heats up SMA rivalry with Novartis
Link: Biopharmadive
 -J Med Chem. 2018 Aug 9;61(15):6501-6517. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). Ratni H et al. Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
Results/Comments: This compound is undergoing pivotal clinical trials and is a promising medicine for the treatment of patients in all ages and stages with SMA
Link: Abstract  
 spacer
 spacer
ProQR announces positive top-line results from the phase 1/2 study of Sepofarsen in LCA10 patients
ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, announced on October 10th positive top-line results from the PQ-110-001 study, a Phase 1/2 dose range finding, first-in-human trial of sepofarsen (QR-110) in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene. Based on positive 3-month interim results from the Phase 1/2 trial (Nature Medicine 2018) the Phase 2/3 Illuminate trial was initiated earlier this year. The 12-month top-line results from the Phase 1/2 trial that will be presented today confirm durable activity of sepofarsen for up to one year in patients with LCA10.
For further information 
 spacer
 spacer
Related Informations / Publications
-OCT 2019: ProQR to Present Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients ahead of AAO
Results/Comments: PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 - 17 years) and 6 adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients are receiving four intravitreal injections of sepofarsen into one eye; one injection every three months, with the other eye remaining untreated
Link: Press Release
 -AUG 2019: ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)
Results/Comments: PQ-1123-001, is a first-in-human study that will initially include up to 12 adults with adRP due to the P23H mutation in in the rhodopsin (RHO) gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in which a single intravitreal injection of QR-1123 or sham procedure will be given in one eye
Link: Press Release
-Nat Med. 2019 Feb;25(2):225-228. Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect. Cideciyan AV et al. Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA Results/Comments: There were no serious adverse events, and vision improved at 3 months. The visual acuity of one exceptional responder improved from light perception to 20/400 . Link: Abstract  
 spacer
 spacer
Ionis' Factor XI anti-thrombotic medicine advances with Bayer following positive clinical results
Ionis Pharmaceuticals, a leader in RNA-targeted therapeutics, announced on October 09th that the company has been notified by its partner Bayer about the decision to advance IONIS-FXI-LRx following positive clinical results. IONIS-FXI-LRx is an antisense medicine being developed to treat patients with clotting disorders. ONIS-FXI-LRx utilizes Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform and is designed to reduce the production of Factor XI (FXI), a clotting factor produced in the liver. High levels of FXI increase the risk of thrombosis and can be responsible for heart attacks and strokes. Alternatively, individuals deficient in FXI have a lower incidence of thrombosis-related events and little to no increase in bleeding risk. For further information 
Related Informations / Publications
-Semin Thromb Hemost. 2019 Jul;45(5):502-508. The Safety and Efficacy of Novel Agents Targeting Factors XI and XII in Early Phase Human Trials. DeLoughery EP et al. Mayo Clinic School of Medicine, Mayo Clinic, Rochester, Minnesota, USA Results/Comments: Though still far from the marketplace, drugs targeting FXI and FXII have the potential to usher in a new era of anticoagulation therapy
 Link: Abstract
 -FEB 2017: Ionis Earns $75 Million from Bayer for Advancing IONIS-FXI Rx and IONIS-FXI-L Rx
Results/Comments: Under the agreement, Ionis plans to conduct a Phase 2b study evaluating IONIS-FXIRx in approximately 200 patients with end-stage renal disease on hemodialysis to finalize dose selection. Additionally, Ionis plans to rapidly develop IONIS-FXI-LRx through Phase 1. Link: PRNewswire  
 spacer
 spacer
 spacer
m&A - AGREEMENTS
Akcea and Pfizer announce licensing agreement for investigative antisense therapy AKCEA-ANGPTL3-L(Rx)
Akcea Therapeutics, a majority-owned affiliate of Ionis Pharmaceuticals, and Pfizer announced on October 07th that the companies have entered into a worldwide exclusive licensing agreement for AKCEA-ANGPTL3-LRx, an investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases. AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver, a key regulator of triglycerides, cholesterol, glucose and energy metabolism. AKCEA-ANGPTL3-LRx is currently being evaluated in a Phase 2 study in patients with Type 2 diabetes, hypertriglyceridemia and non-alcoholic fatty liver disease (NAFLD). Under terms of the agreement, Akcea and Ionis will receive a $250 million upfront license fee, which will be split equally between the two companies. Akcea will settle its $125 million obligation to Ionis in Akcea common stock. The companies are also eligible to receive development, regulatory and sales milestone payments of up to $1.3 billion and tiered, double-digit royalties on annual worldwide net sales following marketing approval of AKCEA-ANGPTL3-LRx. Future milestone payments and royalties will be split equally between Akcea and Ionis. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the ongoing Phase 2 study. Prior to regulatory filing for marketing approval, Akcea has the right, at its option to participate in certain commercialization activities with Pfizer in the U.S. and certain additional markets on pre-defined terms and based on meeting pre-defined criteria.
For further information  
Related Informations / Publications
-SEP 2019: Positive Phase 1 Results of AKCEA-TTR-LRx Presented at the Heart Failure Society of America Annual Meeting. Link: Press Release
-AUG 2019: New England Journal of Medicine Publishes Results from Pivotal Study of Waylivra® (volanesorsen) in Patients with Familial Chylomicronemia Syndrome
Link: NEJM - Press Release
 -DEC 2017: Akcea Initiates Phase 2 Study of AKCEA-ANGPTL3-LRx in Patients with Hypertriglyceridemia, Type 2 Diabetes Mellitus and Nonalcoholic Fatty Liver Disease (NAFLD). Link: Press Release
-N Engl J Med. 2017 Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides. Jul 20;377(3):222-232. Graham MJ et al. Akcea Therapeutics, Cambridge, MA, USA
Results/Comments: Oligonucleotides targeting mouse Angptl3 retarded the progression of atherosclerosis and reduced levels of atherogenic lipoproteins in mice.
Link: Abstract - Full Text    
Editas Medicine and MaxCyte announce clinical and commercial license agreement for engineered cell medicines
Editas Medicine and MaxCyte, a cell-based therapies and life sciences company, announced on October 07th a new clinical and commercial license agreement. Editas Medicine will use MaxCyte’s Flow Electroporation® technology and ExPERT™ instruments for the advancement of engineered cell medicines, including EDIT-301, an experimental CRISPR medicine designed to durably treat sickle cell disease .and beta-thalassemia. Under the terms of the agreement, Editas Medicine obtains non-exclusive clinical and commercial use rights to MaxCyte's cell engineering platform to develop up to five therapies including four immuno-oncology therapies, and in return MaxCyte will receive development and approval milestones and sales-based payments in addition to other licensing fees. For further information 
 spacer
Related Informations / Publications
-JUL 2019: Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10
Results/Comments: The Brilliance clinical trial is a Phase 1/2 study to evaluate AGN-151587 for the treatment of LCA10. The study will assess safety, tolerability, and efficacy in approximately 18 patients. Link: Press Release
-JUN 2019: Editas Medicine Presents Pre-Clinical Data for Treatment of Sickle Cell Disease and Beta-Thalassemia at the 24th Congress of the European Hematology Association Results/Comments: Results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer (BC11Ae) and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia. Link: Press Release
-MAY 2019: MaxCyte Progresses Phase I Clinical Trial of Lead mRNA-based Cell Therapy from its CARMA™ Platform Link: Press Release
-Mol Ther Methods Clin Dev. 2018 Jun 15;10:105-112. Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population. Simhadri VL et al. Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA. Link: Abstract - Full Text    
 spacer
PTC Therapeutics establishes strategic collaboration with Aldevron to support GMP plasmid manufacturing
PTC Therapeutics announced on October 07th a collaboration with Aldevron to secure GMP manufacturing capacity for PTC's gene therapy portfolio, including the Friedreich ataxia and Angelman syndrome programs in PTC's growing gene therapy pipeline. The central nervous system (CNS) gene therapy platform at PTC uses targeted micro-dosing applied directly to areas involved in the disorder. This allows for greater efficacy, durability, lower risk of immunogenicity or other off-target effect, and efficient and scalable manufacturing. PTC has an advancing gene therapy pipeline for CNS disorders, including programs for AADC deficiency, Friedreich Ataxia and Angelman Syndrome. PTC is preparing a BLA for the treatment of AADC deficiency for submission to the FDA in the fourth quarter of 2019. For further information 
Related Informations / Publications
-JUL 2019: EQT to Acquire Majority Interest in Aldevron - the Leading Global Supplier of Plasmid DNA Used in Cell and Gene Therapies
Results/Comments: EQT will support Aldevron through investments in additional production capacity, R&D, and growth initiatives and by leveraging EQT's strong healthcare expertise, global presence, and network of Industrial Advisors
Link: PRNewswire
-JUN 2019: Biologics company Aldevron unveils expansion of its North Dakota campus Link: Fiercepharma
-The CRISPR Journal, June 2019, Vol. 2, No. 3. Aldevron Expands to Facilitate Emerging Therapies. Link: Abstract   
Lonza agrees to manufacture Prevail Therapeutics’ novel AAV-based gene therapies for patients with neurodegenerative diseases
Prevail Therapeutics, a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, and Lonza announced on October 8th that the companies have entered into a strategic collaboration. Lonza and Prevail have been working together since 2018, with an initial focus on process development, towards the GMP manufacturing of Prevail's two lead programs, PR001 and PR006. Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail's pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases at its gene therapy center of excellence in Houston, TX. Prevail's pipeline includes PR001, an AAV9-based gene therapy delivering GBA1, in development for Parkinson’s disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease patients (nGD). Prevail plans to initiate a Phase 1/2 clinical trial of PR001 in PD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN mutation (FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration also has the potential to extend to Prevail's future pipeline of AAV-based gene therapy programs. For further information
Related Informations / Publications
-SEP 2019: Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease
Results / Comment: Prevail is also developing PR001 for pediatric nGD, a second indication with urgent unmet need. The company submitted an IND to the FDA for PR001 for the treatment of pediatric patients with nGD. Link: Press Release
 -JUN 2019: Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations
Link: Abstract
-Pharmacol Res. 2019 Apr 23:104240. Pharmacology of nerve growth factor and discovery of tanezumab, an anti-nerve growth factor antibody and pain therapeutic. Hefti F. Prevail Therapeutics Inc., 430 East 29th Street, New York, NY, 10016, USA
Link: Abstract
Cellectis and Lonza enter cGMP manufacturing service agreement for Cellectis’ allogeneic UCART product candidates
Cellectis, a clinical-stage biopharmaceutical company focused on developing immunotherapies based on allogeneic gene-edited CAR T-cells (UCART), and Lonza announced on October 01st that the companies have entered into a manufacturing service agreement covering clinical manufacturing of Cellectis’ allogeneic UCART product candidates targeting hematological malignancies. Lonza is in charge of implementing Cellectis’ manufacturing processes as per current Good Manufacturing Practices (cGMP) in a way that meets the highest quality and safety standards outlined by the FDA. The manufacturing will take place at Lonza's GMP facility in Geleen, Netherlands. Lonza's supply will complement Cellectis' ongoing collaboration and in-house manufacturing sites, IMPACT and SMART, which are currently under construction. The manufacturing process of Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, non-alloreactive engineered CAR T-cells. UCARTs are intended to be readily available CAR T-cells for a large patient population. Their production is industrialized with defined pharmaceutical release criteria. For further information
 spacer
 spacer
Related Informations / Publications
-JUL 2019: Cellectis Publishes New CAR Design to Control CAR T-Cells in Non-Lethal Way Link: Press Release
-MAR 201 : Cellectis Enters Lease Agreement to Build Manufacturing Facility, Advancing Towards Commercialization of its UCART Portfolio
Results/Comments: These new manufacturing plants will allow GMP manufacturing for both clinical supplies and commercial products according to the FDA and EMA guidelines, and will be fully equipped to support a potential regulatory approval.
Link: Press Release    
Alexion and Stealth announce agreement for option to co-develop and commercialize late-stage therapy for mitochondrial diseases
Alexion Pharmaceuticals and Stealth BioTherapeutics announced on October 10th an agreement for an option to co-develop and commercialize elamipretide for mitochondrial diseases. Currently being evaluated in a Phase 3 study in people with primary mitochondrial myopathy (PMM) - a genetic mitochondrial disease - elamipretide is a novel, potential first-in-class therapy that targets mitochondrial dysfunction. There are currently no therapies approved to treat PMM, which is characterized by debilitating skeletal muscle weakness, chronic fatigue and exercise intolerance. Under the terms of the agreement, Alexion will have the opportunity to exercise the option following the delivery of results from the Phase 3 study currently underway in PMM.
For further information 
Related Informations / Publications
-AUG 2019: People with Mitochondrial Disease Have Significant Medical Costs Results/Comments: A recent study published in Orphanet Journal of Rare Diseases reported that people with mitochondrial disease (mito) have significant medical costs, higher than that of the general population, and similar to a person with multiple sclerosis (MS) or amyotrophic lateral sclerosis (ALS). Link: Press Release
 -Cell Rep. 2018 Mar 13;22(11):2818-2826. Optimized Mitochondrial Targeting of Proteins Encoded by Modified mRNAs Rescues Cells Harboring Mutations in mtATP6. Chin RM et al. Alexion Pharmaceuticals, Inc., 100 College Street, New Haven, CT 06510, USA
Results/Comments: Rechearchers synthesized chemically modified mRNAs that encode for an optimized allotopic expression construct for mtATP6. These mRNAs were able to functionally rescue a cell line harboring the 8993T > G point mutation in the mtATP6 gene. Link: Abstract  
TrakCel announces strategic partnership with WuXi AppTec Advanced Therapies for optimization of supply chain for cell and gene therapy treatments
TrakCel, a software developer for cell and gene therapy supply chain tracking and orchestration systems and WuXi AppTec Advanced Therapies, a Contract Development and Manufacturing Organization (CDMO), announced on October 01st a collaborative agreement designed to accelerate the seamless delivery of advanced therapy treatments to bring an end-to-end solution to mutual customers. To provide full visibility and control of their collection-to-administration supply chain, cell therapy companies need to ensure consistent product handling and data capture across all supply chain partners. This is increasingly being achieved through the deployment of supply chain management software, such as TrakCel’s platform. Challenges can arise if software-driven workflows do not account for established processes in place at each supply chain partner. For example, if a contract manufacturer needs to change their processes to fit in with those designed into supply chain management software, this can increase costs and timelines for software adoption. Conversely, rewriting software after it becomes apparent that it will not work for a contract manufacturer also incurs additional expense and can delay software deployment. For further information 
Related Informations / Publicationss
-SEP 2019: McKesson and TrakCel Launch Innovative Collaboration to Support Commercialization of Cell and Gene Therapies
Link: Press Release  
 spacer
 spacer
 spacer
 spacer
Please, add our e-mail (alb@biopharmanalyses.fr) to your e-mail address book.
To learn more about us: 
 spacer
LinkedIn BioPharmAnalyses
LinkedIn Anne-Lise Berthier
Twitter
 spacer
 spacer
Open in a browser/Ouvrir dans un navigateur
To unsubscribe from all further electronic notices from BioPharmAnalyses please e-mail your name to alb@biopharmanalyses.fr entering "Unsubscribe" in the subject line or click Unsubscribe/Se désinscrire
Click here to print this newsletter.

© BioPharmAnalyses. 
 spacer