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Newsletter September 2019

News: Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many of those problems by packing a gene-editing payload into a tiny, customizable, synthetic nanocapsule. They described the delivery system and its cargo today (Sept. 9, 2019) in the journal Nature Nanotechnology.

News: Gene Therapy Developers Issue Principles for Human Genome Editing
The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology. While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.

Company: Genemedi
Biotechnology company ( with long standing expertise in AAV production, AAV vector optimizion, capsid evolution and new AAV generation. Founded in 2010, Genemedi is a technical service company focusing on the research and development of AAV, adenoviral and lentiviral productions. We strive to make contributions to the development and clinical transformation of gene therapy technology, which is important for the whole human being.

Conference: 9th annual Partnership Opportunities in Drug Delivery
The 9th annual Partnership Opportunities in Drug Delivery (PODD) conference, 7-8 October 2019 in Boston, is a business development event covering the latest advances in drug delivery science, while also providing a meeting place to network and form new partnerships. The PODD event features a wide range of innovative drug delivery technologies that could potentially improve the delivery of various types of drugs, including proteins, peptides, oligonucleotides, biologics, and small molecules.
Register here...

Conference: Macrophage-directed Therapies Summit
The inaugural Macrophage-directed Therapies Summit, 22-24 October 2019 in Boston, is the only platform dedicated to helping leaders from pharma, biotech and academic institutes to successfully achieve clinical proof of concept when translating macrophage therapies into the clinic whilst ensuring safety, control and effective combination therapies. Join your macrophage community and discuss strategies to characterize macrophage biology to understand their role in tumor progression and improve targeting precision to prevent off-tumor toxicities.
Register here...

Conference: 5th Annual Genome Editing Congress
Over 600 senior-level delegates representing internationally renowned research & academic institutions, clinical research institutions and pharmaceutical companies. Over 20 case studies and presentations focused on the latest developments in gene editing techniques and case studies on the therapeutic applications of genome editing technologies. The 5th Annual Genome Editing Congress takes places from 7-8 November 2019 in London. Including 2 Interactive Streams:
• Genome Editing Techniques and Technologies
• Therapeutic Applications of Genome Editing

Register here...

Conference: World Orphan Drug Congres
The World Orphan Drug Congress and Cell & Gene Therapy, 12-14 November 2019 in Barcelona, is the meeting place for the rare disease community. The agenda covers the strategic and commercial aspects of bringing new treatments to patients who suffer from rare diseases. Government authorities, payers, industry and patient advocacy groups are all joining us. You can also meet biotech start-ups and investors who are showcasing their orphan drug technology and pipeline.
Register here...

Conference: 3rd Annual Ophthalmic Drugs Conference
SMi Group are pleased to present the 3rd Annual Ophthalmic Drugs Conference and Focus Day, taking place on the 18-20 November 2019 in London. The global ophthalmic drug market size is expected to reach a value of $30 billion USD by 2023 with growing focus on alternative delivery approaches to replace intravitreal drug delivery and novel drug development being the key factors driving the market.
With this in mind, this year’s Ophthalmic Drugs conference will explore new discoveries in the treatment of ocular rare disease; innovations in gene therapy; the challenges in drug delivery through a complex barrier; patient comfort and regulatory compliance which make up core components within the Ophthalmic Drug sphere.

Register here...

- 7 - 8 October 2019, 9th annual Partnership Opportunities in Drug Delivery, Boston, MA
- 9 October 2019, The Cell & Gene Therapy Strategy Meeting Europe, Zurich, Switzerland
- 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
- 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
- 20 - 24 January 2020, PepTalk event on Cell and Gene Therapies, San Diego, CA
- 5 - 6 February 2020, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 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
- 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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