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Newsletter October 2019

News: World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results
At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. Weinstein, whose team first administered the investigational gene therapy at UConn John Dempsey Hospital in Farmington, Connecticut, on July 24, 2018, calls the results “remarkable.”

Conference: 3rd Complement-based Drug Development Summit
The Complement-based Drug Development Summit, 13-15 November 2019 in Boston, is the only industry dedicated meeting that brings you the most up to date clinical and commercial developments utilizing complement inhibitors in rare and common disease indications. You will gain key insight on overcoming end to end challenges from preclinical modelling to streamlining process development. This event will enable you to understand the fundamental role of complement in specialized cells to manipulate the complement cascade to treat common and rare diseases. Join experts from InflaRx, Apellis, Novartis, Roche, Genentech and Achillion to discuss targeting the classical, lectin and alternative pathways and creating robust testing and assay measures for patient selection and response. With over 150 of the industry’s pioneers we will gather to advance to the clinic novel complement based drugs beyond C5.
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Conference: 1st RNA Editing Summit
As CRISPR genome editing struggles to overcome key specificity and delivery challenges in its pursuit to realize therapeutic applications, the 1st RNA Editing Summit 2019, 19-20 November 2019 in Boston, accelerates the translation and commercialization of RNA editing for an array of research and therapeutic applications. As the therapeutic potential of RNA editing explodes, discover the fundamental mechanisms of RNA editing, such as recruiting endogenous ADARs with encodable guideRNAs, and learn about the novel approaches being developed to precisely and efficiently deliver intended RNA edits. With the temporary nature of RNA editing providing a huge opportunity in many areas beyond genetic diseases, uncover how to recruit endogenous editing enzymes and ultimately deliver safe and effective transcriptome edits. Join RNA, functional genomic, cell biology and immuno-oncology experts from large pharma, biotech and academia at the 1st RNA Editing Summit 2019 in the pursuit of translating emerging RNA editing mechanisms into therapeutic reality.
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Conference: 2nd Annual RNA-Targeted Drug Discovery Summit
Finally drug the ‘undruggable’ RNA! Join 100+ of your colleagues from large pharma, innovative biotech and KOLs of academia at the 2nd RNA- Targeted Drug Discovery Summit, 2-4 December 2019 in Boston to discuss unique discovery and development challenges that must be overcome for novel RNA targeted small molecule therapeutics to achieve their seemingly limitless potential. As a Gene Therapy Net subscriber, use your unique 10% discount code: GTN10.
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Conference: Gene Therapy for Neurological Disorders
Discover Novel Gene Therapy Delivery Routes to Effectively Target the CNS, Optimizing Preclinical Models to Confidently Translate into the Clinic & Mitigate Immunogenicity to Deliver an Efficacious Dose. Built with the field’s thought-leaders from the likes of Pfizer, Biogen,Voyager and Novartis, GTxN is the industry’s first and only meeting dedicated to solving your translational drug development challenges enabling you to accelerate the development of your neurological gene therapy candidate. Join 100+ of your peers at this definitive conference (Gene Therapy for Neurological Disorders Summit,GTxN, 10-12 December in Boston) over 3 days, 2 interactive workshops, 6+ hours of dedicated networking time and 18 expert industry speakers.
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Company: Batavia Biosciences B.V.
Batavia Biosciences is a contract development and manufacturing organization, specialized in vaccines and gene therapies. To accelerate the journey to the clinic, we offer a full range of development and manufacturing services for all major classes of biopharmaceutical, and have developed a portfolio of technologies aimed to improve product yield, reduce process development time and increase product stability. These technologies have demonstrated to reduce risks, improve timelines and reduce costs in biopharmaceutical development to make sure the product receives the patient without any unnecessary delays. We are an active player in Global Health, where we collaborate with prominent academic institutions and public organizations to improve the availability and affordability of key biopharmaceuticals for all in need.

- 15 – 16 October 2019, 9th Annual Orphan Drugs and Rare Diseases conference, London, UK
- 15 – 17 October 2019, Gene Therapy for Rare Disorders Europe 2019, London, UK
- 21 – 22 October 2019, NORD's 2019 Rare Diseases & Orphan Products Breakthrough Summit, Washington, D.C.
- 21 – 25 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain
- 22 – 24 October 2019, Macrophage-directed Therapies Summit, Boston, MA
- 23 – 25 October 2019, Neuromuscular Drug Development Summit - Gene Therapies, Boston, MA
- 24 – 25 October 2019, 5th Annual Cell & Gene Therapy Congress, London, UK
- 29 – 31 October 2019, Cell and Gene Therapy Manufacturing Forum event, London, UK
- 7 – 8 November 2019, 5th Annual Genome Editing Congress, London, UK
- 12 – 14 November 2019, World Orphan Drug Congress, Barcelona, Spain
- 13 – 15 November 2019, 3rd Complement-based Drug Development Summit - Gene Therapy, Boston, MA
- 19 – 20 November 2019, 1st RNA Editing Summit, Boston, MA
- 19 – 20 November 2019, 3rd Annual Ophthalmic Drugs Conference - gene therapy for eye diseases, London, UK
- 2 – 4 December 2019, 2nd RNA-Targeted Drug Discovery Summit, Boston, MA
- 10 – 12 December 2019, Gene Therapy for Neurological Disorders, Boston, MA
- 20 - 24 January 2020, PepTalk event on Cell and Gene Therapies, San Diego, CA
- 29 - 30 January 2020, CAR-T Congress EU, London, UK
- 29 - 30 January 2020, Vein-to-Vein Congress EU - Manufacturing, scale-up, regulatory and logistical bottlenecks to commercialise cell therapies, London, UK
- 5 - 6 February 2020, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 22 - 24 February 2020, CAR-TCR Summit Europe, London, UK
- 2 - 3 March 2020, Tri-Conference 2020 - Renaissance of Gene Therapy and Genome Editing, San Francisco, CA
- 23 - 25 March 2020, International Society for BioProcess Technology 10th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 30 March - 2 April 2020, 4th Annual Gene Therapy for Rare Disorders, Boston, MA
- 31 March - 2 April 2020, 15th Annual World Advanced Therapies Congress, London, UK
- 29 - 30 April 2020, CAR-T Congress USA, Boston, MA
- 12 - 15 May 2020, American Society of Gene and Cell Therapy (ASGCT) 23th Annual Meeting, Boston, MA
- 15 - 19 June 2020, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia
- 20 – 23 October 2020, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK


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