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Newsletter May 2023

News: Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS)

Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS) is an ultra rare disease caused by a mutation in the gene ACTA2 that results in a malfunction of the smooth muscle cells in the whole body. Fixed and congenital dilated pupils, patent ductus arteriosus/aorto pulmonary window and other complications related to the bladder, lungs and guts, are the common manifestations of this mutation with only 60 diagnosed people in the world, most of them children. Join the 2023 MSMDS Conference taking place May 5-7 in Boston and online.

Conference: World Orphan Drug Congress USA 2023

The World Orphan Drug Congress USA attracts the entire rare disease ecosystem – breaking down silos and fostering collaboration to help rare disease patients.

The leading orphan drug pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers rely on the congress to convene and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.

Join the most comprehensive program and inclusive gathering of rare disease stakeholders for 50% off with Gene Therapy Network’s exclusive code GTN50.

Get ready to join over 2000+ attendees, from 50+ countries, outside of Washington D.C. this May 23-25.

Conference: 5th Treg Summit

Join Treg trailblazers as they come together to recognize therapy efficacy, determine safety and translate the next-generation of therapies into the clinic! 5th Treg Summit, May 30 - June 1.

Conference: Next Gen Inhalation Delivery Summit

This June, the hotly anticipated Next Gen Inhalation Delivery Summit (June 20-22) will debut, where we will unite 80+ senior leaders at this definitive meeting dedicated to the inhalation community. Whether your work in COPD, asthma, IPF, lung cancer or infectious disease, this comprehensive forum offers the solution to capitalize on the common goal of targeted lung delivery.
Be part of the conversation in Boston this June, where we unpick:
- Overcoming pulmonary biological barriers to minimize dosage and maximize therapeutic benefit
- Advancing your inhalation formulation techniques for improved stability and longer shelf-lives
- Ensuring device-formulation compatibility with patient compliance and patient convenience
With key speakers from the likes of Janssen, Avalyn Pharma, and Pieris, you cannot afford to miss out!

Conference: 2nd Annual Optimizing AAV Safety Summit

Predict & Monitor AAV Safety Signals to De-Risk Genetic Therapy Development.
With more than 300 trials in preclinical and clinical development actively using AAV as their vector of choice, the priority for any company working in this space is to ensure that each and every one of those therapies is the safest version it can be.
In this context, the 2nd Annual Optimizing AAV Safety Summit is returning in 2023 (July 11-13), bringing together genetic therapy leaders across non-clinical development, clinical development, toxicology, biodistribution, and immunology who are all working to optimize the safety of their AAV-based drug products in their day-to-day.
By addressing key topics including how to extract the most relevant data from preclinical studies to better inform how humans will respond to an AAV-based therapy in the clinic, analyzing potential integration risks of AAV serotypes, and investigating regulatory approaches to safety packages for submission, this meeting will inform how to streamline AAV safety processes to de-risk delays and shorten timelines to approval.
Join us in Boston to be part of the solution to bringing safer, effective genetic therapies to patients and gain immediately implementable insights to sharpen your programs currently in preclinical and clinical development.
Register now…

Conference: 3rd Tumor Myeloid-Directed Therapies Summit

The 3rd Annual Tumor Myeloid-Directed Therapies Summit (July 18 - 20) returns with over 75% new speakers to share insights across a range of myeloid targets, including CD40, CD39, LILRB2, CLEC-1 as well as CAR-M therapies. The one-stop shop continues to be your go-to forum to accelerate myeloid therapy progression by determining optimal combination strategy, optimize translational studies, discover novel targets, and guarantee patient safety. Connect with this up-and-coming community to reinvent targeting and harness the untapped potential of the myeloid.

Conference: 3rd Annual mRNA-Based Therapeutics Summit

Uniting industry experts from the likes of the FDA, Pfizer, Moderna, BioNTech, Sanofi and more, the 3rd mRNA-Based Therapeutics Summit returns to Boston this July as your mRNA one-stop-shop – showcasing exclusive new data and insights from the leaders in the field (July 26-28). Hear from 100+ world-class speakers from across large pharma, innovative biotechs and academia across 5 dedicated content tracks – catering to discovery, translation, clinical development, CMC, and logistics.

With 200+ clinically active mRNA drugs and a further 500+ in preclinical development, this meeting is a must-attend this summer to advance towards the clinic and deliver the promise of better mRNA medicines.

Conference: Drug Discovery and Development

Drug Discovery and Development (21st September) is an annual conference that aims to bring together key decision makers such as CXOs, Vps, Directors, Heads, Scientists, and Researchers [event's target from all over Europe]. This year, we expect 150+ attendees to participate and would be honored to have you as one of our valued speakers.
Our Event will include 15+keynotes, a mix of Customer Case Studies, Research Projects, and Keynotes presentations.

Conference: mRNA Analytical Development Summit Europe

Uniting technical mRNA specialists in Europe for the very first time, the inaugural mRNA Analytical Development Summit Europe (29th - 31st August) will bring 80+ Analytical Development, CMC, Quality, and Regulatory experts to Berlin this August. See the brochure: https://ter.li/wd458g
Highlighting the importance of advanced analytical approaches and adherence to regulatory requirements, this meeting is the only industry-dedicated forum to showcase critical expertise and the latest techniques in optimised analytics for leading European biopharma.
Hear from 19+ European thought leaders across 3 days and 2 workshops of immersive content, with speakers confirmed from AstraZeneca, Ziphius Vaccines, BioNTech, Moderna, GSK, VaxEquity, Novartis and more.
This meeting will enable you to confidently identify and quantify critical quality attributes through advanced analytical platforms to guarantee the quality, efficacy and safety of your mRNA therapeutics and vaccines, safeguarding your regulatory submissions in 2023 and beyond.
Register now…

Upcoming conferences and meetings

16 - 18 May 2023 - 2nd Next Generation RNA Therapeutics Summit - Boston, MA
17 - 20 May 2023 - American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting - Los Angeles, CA
22 - 25 May 2023 - 5th Annual Allogeneic Cell Therapies Summit - Boston, MA
23 - 24 May 2023 - Global Biologics Cell and Gene Legal Summit - Brussels, Belgium
23 - 25 May 2023 - World Orphan Drug Congress USA 2023 - Washington, D.C.
26 - 28 May 2023 - Symposium on World Cell and Molecular Biology 2023 - Singapore
30 May - 1 June 2023 - 5th Treg Summit - Boston, MA
30 May - 1 June 2023 - 7th R&D Sourcing & Procurement in Pharma Summit - Boston, MA
31 May - 2 June 2023 - 2nd Cell Therapy Potency Assay Summit - Boston, MA
6 - 8 June 2023 - Gene Therapy for Neurological Disorders Summit Europe - Amsterdam, The Netherlands
7 - 8 June 2023 - Gene Therapy CMC & Analytics - Online event
12 - 13 June 2023 - Annual meeting of the Netherlands Society of Gene & Cell Therapy - Lunteren, The Netherlands
13 - 15 June 2023 - 3rd Gene Therapy Patient Engagement Summit - Boston, MA
20 – 21 June 2023 - 3rd Gene and Cell Therapies: CMC, CGT & Vector Manufacturing Summit - Vienna, Austria
20 – 22 June 2023 - Next Gen Inhalation Delivery Summit - Boston, MA
21 – 22 June 2023 - 6th Aseptic Processing Summit - Manufacturing processes for ATMPs and vaccines - Vienna
26 - 29 June 2023 - Gene Therapy & Rare Diseases conference - Boston, MA
27 - 29 June 2023 - Next Generation Gene Therapy Vectors Summit - Boston, MA
11 - 13 July 2023 - 4th RNA Editing Summit - Boston, MA
11 - 13 July 2023 - 2nd Annual Optimizing AAV Safety Summit - Boston, MA
18 - 20 July 2023 - 3rd Tumor Myeloid-Directed Therapies Summit - Boston, MA
26 - 28 July 2023 - 3rd Annual mRNA-Based Therapeutics Summit - Boston, MA
9 August 2023 - Drug Discovery and Development - Cell & Gene Therapies - London, UK
14 - 17 August 2023 - The Bioprocessing Summit 2023 - Boston, MA
29 - 31 August 2023 - 3rd Inner Ear Disorders Therapeutics Summit - Gene & Cell Therapies - Boston, MA
29 - 31 August 2023 - mRNA Analytical Development Summit Europe - Berlin, Germany
29 August - 1 September 2023 - 8th CAR-TCR Summit - Boston, MA
30 - 31 August 2023 - Viral Vector Formulation & Process Development Summit Europe - Amsterdam, Netherlands
5 - 7 September 2023 - 5th Exosome Based Therapeutic Development Summit - Gene Therapies - Boston, MA
6 - 7 September 2023 - RNA Leaders USA Congress - Boston, MA
18 - 21 September 2023 - Cell & Gene Therapy Manufacturing & Commercialization US - Boston, MA
18 - 21 September 2023 - BioProcess International - ATMP - Boston, MA
27 - 29 September 2023 - Smart Bioprocessing & Pharma 4.0 Summit - Cell & Gene Therapies - Berlin, Germany
18 – 19 October 2023 - World Drug Safety Congress Americas 2023 - Boston, MA
24 – 27 October 2023 - Annual Congress of the European Society of Gene and Cell Therapy - Brussels, Belgium

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