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Newsletter May 2021

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News: New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
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Conference: 3rd Annual Blood-Brain Barrier Summit
At the 3rd Blood-Brain Barrier Summit,28-30 June 2021, we will uncover how leading biopharma and academics are working to enhance blood-brain barrier penetration using antibody therapies, nanoparticle engineering, AAV capsids, focused ultrasound, extracellular vesicles and more!
We have collated a comprehensive variety of topics to showcase the latest scientific advances and evaluate new and innovative approaches in this very competitive field, with expert speakers from AbbVie, Roche, Novartis, AskBio, Ossianix and more.
Join this intimate digital forum to be part of the conversation focused on enhancing drug delivery across the BBB and network to build meaningful partnerships with over 80 BBB specialists.

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Conference: Epigenetic Therapeutic Targets Summit
Returning for its second year, the Epigenetic Therapeutic Target Summit, 13-15 July 2021, is the first-ever industry forum dedicated exclusively to the frontier of drug development in epigenetics. The two-day program will enable you to explore the full spectrum oncological and non-oncological applications of genetic regulation therapeutics that is being investigated to identify new unmet clinical needs that epigenetics could be central to treating.
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Conference: mRNA-Based Therapeutics Summit
The mRNA therapeutics summit, 13-15 July 2021, represent a disruptive class of medicines with the ability to make global change. Offering a fast and effective drug against COVID-19, mRNA vaccines have provided relief around the world, setting out an exciting roadmap for potential applications of mRNA. Despite this, mRNA therapeutic development exists not without challenges and the inaugural mRNA-Based Therapeutics Summit will ensure this important class of medicines continues to strive for success.
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Conference: 2nd Gene Therapy for Neurological Disorders Europe
Returning digitally in 2021, the 2nd Annual Gene Therapy for Neurological Disorders Europe, 19-21 July 2021, is your chance to discover the latest neurological targets, familiarise yourself with the European landscape, and debate and discuss with the leading minds of the field from the likes of UCB, Lysogene, UniQure and AskBio, as they seek to harness the opportunity and move from rare to common disorders such as Alzheimer’s, Parkinson’s and epilepsy.
Focusing on translation and ‘what’s new’ in the gene therapy for neurological disorders field, this year’s 3-day agenda has been developed alongside neurological experts from Handl Therapeutics, Novartis, Spark Therapeutics and many more to capture the most pressing challenges and new opportunities facing the field.
Don’t miss your opportunity to benchmark against your competitors, hear the state of the art technology advances and research in the field, and make new connections at the last digital European meeting, to revolutionise the development and delivery of your neurological gene therapy.

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Conference: Gamma Delta T Therapies
With race to market for the first allogeneic and solid tumor targeting cell immunotherapy well and truly on, the explosive excitement in Gamma Delta T Therapies, 27-29 July 2021, continues to increase, as we begin to see the early clinical data read out on safety and efficacy.
Supercharging development of Gamma Delta T Therapies to bring lifesaving and cost-effective solid tumor treatments to patients in need, the Annual Gamma Delta T Therapies Summit returns online as a 3 day virtual event gathering the world’s leaders who are harnessing GDT cells to bring to market the first persistent, safe and effective cell therapy for solid tumors.
Join the likes of Adicet bio, IN8 bio, Gamma Delta Therapeutics, LAVA Therapeutics, Imcheck Therapeutics and many others and save 10% off your attendance with the code ‘GDTG10’.

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Conferences
- 18 - 20 May 2021, Genetic Vaccine Development for Infectious Diseases Summit, Online event
- 20 - 21 May 2021, 5th Annual MarketsandMarkets Bioprocessing of Biotherapeutics-UK,EU & APAC, Online event
- 25 - 27 May 2021, Inner Ear Disorders Therapeutics Summit - Gene Therapy Solutions, Online event
- 25 - 27 May 2021, Gene Therapy Analytical Development Europe, Online event
- 25 - 27 May 2021, Gene Therapy for Muscular Disorders, Online event
- 26 - 28 May 2021, International Society for Cell and Gene Therapy (ISCT) New Orleans virtual meeting, Online event
- 8 - 10 June 2021, Next Generation Gene Therapy Vectors, Online event
- 15 - 17 June 2021, 2nd RNAi- Based Therapeutics Summit, Online event
- 28 - 30 June 2021, 3rd Annual Blood-Brain Barrier Summit - Exploring CNS Gene Therapy & New Generation Vectors, Online event
- 13 - 15 July 2021, Epigenetic Therapeutic Targets Summit, Online event
- 13 - 15 July 2021, mRNA-Based Therapeutics Summit, Online event
- 19 - 21 July 2021, 2nd Gene Therapy for Neurological Disorders Europe, Online event
- 27 - 29 July 2021, Gamma Delta T Therapies Summit, Online event
- 3 - 5 August 2021, 2nd Gene Therapy Immunogenicity Summit, Online event
- 16 - 19 August 2021, 13th Annual The Bioprocessing Summit, Boston, MA
- 25 - 27 August 2021, World Orphan Drug Congress USA, Maryland, USA
- 30 August - 2 September 2021, 6th CAR-TCR Summit, Online event
- 7 - 9 September 2021, Next Generation Cancer Vaccine Development Summit, Online event
- 21 - 22 September 2021, Formulation & Delivery UK: Gene and Cell Therapies, London, UK
- 21 - 23 September 2021, 2nd Gene Therapy for Ophthalmic Disorders, Online event
- 22 - 23 September 2021, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 12 - 13 October 2021, Pharma Europe 2021 - Cell & Gene Therapies, Barcelona, Spain
- 18 - 20 October 2021, Advancing Gene Therapy 2021, Boston, MA
- 19 - 21 October 2021, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Online event
- 27 - 29 October 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 10 - 11 November 2021, Pharma Manufacturing: Gene and Cell Therapies, Online event
- 16 - 17 November 2021, Onco Cell Therapy Summit (OCTS) USA 2021, Boston, MA
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK

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