Alliance for Regenerative Medicine

This month, the sector has seen some notable developments in Brazil. The country granted marketing authorization to Upstaza, a gene therapy developed by PTC Therapeutics, Inc. to treat AADC deficiency. This is the third gene therapy product approved in Brazil, with Novartis’ Zolgensma and Spark Therapeutics, Inc.'s Luxturna both approved in 2020. https://lnkd.in/eE-kvhfE Additionally, GEMMA Biotherapeutics announced last week that it reached a $100 million agreement with Brazil’s Fiocruz, a public health research institute, to develop and manufacture cell and gene therapies for rare diseases. https://lnkd.in/eMH2ynNJ These are encouraging developments for advancing global access to cell and gene therapies beyond regions like North America, Europe, and East Asia.

Looking to relive your favorite moments from ARM's 2024 Cell & Gene Meeting on the Mesa? Most of the conference photos can now be found online through our Flickr account. ?? ?? https://lnkd.in/eMVxQDfU Attendees, speakers, and presenters are welcome to download photos of their favorite moments to share on social media. The photos are organized in albums by each day of the conference. #CGMesa24

As ARM's 2024 Cell & Gene Meeting on the Mesa concluded this week, the #cellandgenetherapy sector saw plenty of activity and attention, driven by some notable headlines. ?? A fresh wave of cell and gene therapy investments and deals ?? In-vivo CRISPR gene editing therapy begins Phase 3 clinical trials ?? BLA submission for gene therapy to treat retinitis pigmentosa planned for 2025 Read the full stories below and subscribe to ARM's Sector Vector newsletter for more weekly news on cell and gene therapy clinical, business, and manufacturing. ?? https://lnkd.in/eD7ZNaU Astellas Pharma and AviadoBio announce exclusive option and license agreement for gene therapy targeting frontotemporal dementia and other disorders. AviadoBio will receive a $20 million equity investment, up to $30 million in upfront payments, and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties. https://lnkd.in/e3CrzXPN Immatics, a developer of adoptive cell therapies to treat cancers, announced the pricing of its initial public offerings and expects to raise approximately $150 million. https://lnkd.in/dF8TD_2i Purespring Therapeutics raised $105 million in Series B financing for its AAV gene therapy programs to treat various kidney diseases. https://lnkd.in/eaSU6qnU Intellia Therapeutics, Inc. announced the initiation of HAELO, a global, pivotal Phase 3 study of its in-vivo CRISPR gene therapy to treat hereditary angioedema. It is Intellia's second in vivo candidate to enter late-stage clinical development. https://lnkd.in/eszSVUDX Nanoscope Therapeutics Inc. announced it plans to submit a BLA in Q1 2025 for its gene therapy to treat Retinitis Pigmentosa. If accepted, it will add to a growing list of CGTs that could receive approval in 2025. https://lnkd.in/gmFv42w2 #CGMesa24

While gene therapy represents a radical shift in our approach to disease treatment, this novel approach poses new challenges for health systems. Last year, ARM and Pfizer?convened a roundtable to discuss advances in gene therapy, current health system barriers, and potential solutions to gene therapy access. This year, we're hosting a panel at the 2024 World Health Summit that will provide an opportunity to share inputs from the roundtable and offer insights into how countries are preparing their health systems for these new innovations. The panel will be held on October 15 at 9:00am UTC. View the full speaker lineup below and learn more about the session here: https://lnkd.in/ek3WDQ-d Moderator Paolo Morgese Alliance for Regenerative Medicine, Vice President, Public Affairs Europe Durhane Wong-Rieger, President and CEO of the Canadian Organization for Rare Disorders and Chair of Rare Disease International Dr. Paul Fennessy, Victoria Department of Health, Former Acting Senior Medical Advisor Jennifer Young, Pfizer, VP and Head, Global Business Policy & Public Affairs Prof. Mauro Marè, Luiss University of Rome, Professor of Finance Sung Hee Choe, Milken Institute, FasterCures MI Health, Managing Director

The past few years have been challenging for capital markets, and the cell and gene therapy sector is no exception. However, ARM data indicates investor interest could be returning. BioSpace highlights insights into the trends shaping the future of investment at ARM's 2024 Cell and Gene Meeting on the Mesa. https://lnkd.in/e3Cv9c8i #CGMesa24

With #CGMesa24 now finished, ARM's Board of Directors has elected our Executive Committee members, including officers, for 2025. Join us in welcoming these distinguished leaders who will play a key role in ARM's leadership for the cell and gene therapy sector. Chair Bob Smith, Venture Partner, OrbiMed Vice-Chair Robert Ang, President and CEO, Vor Bio Secretary Kristin Yarema, Ph.D., CEO, Poseida Therapeutics, Inc. Treasurer Matthew Kapusta, CEO, uniQure Adora Ndu, PharmD, JD, Chief Regulator Officer, BridgeBio Miguel Forte, CEO & Co-Founder, Kiji Therapeutics Laura Sepp-Lorenzino, Ph.D., EVP & CSO, Intellia Therapeutics, Inc.

And that’s a wrap for our 2024 Cell and Gene Meeting on the Mesa! A big thank you to our sponsors, speakers, and partners who made the first year in our new home in Phoenix one to remember. Did you miss out on any panels or workshops you wanted to see? Attendees can rewatch any session through the online conference platform. Also, keep an eye out for more ARM Studios interviews with sector leaders in the coming weeks. ?? Safe travels, and see you next year! ???? #CGMesa24

ARM's 2024 Cell & Gene Meeting on the Mesa is nearly at the finish line. After three action-packed days of 20+ sessions and thousands of partnering meetings, it’s clear from this innovative community that there are countless reasons to believe in the future of #cellandgenetherapy. At the beginning of the conference, ARM CEO Tim Hunt highlighted some of the top reasons why: ?? Promising near-term commercial opportunities ?? Strategic investments by the largest pharma companies; 13 of the top 15 by market cap are in cell and gene therapy ?? Pioneering work in solid tumors and auto-immune disorders (cell therapy) ?? Exciting science will expand patient reach, including more prevalent diseases #CGMesa24

Emerging technologies and societal changes, such as AI and COVID-19, have reshaped how companies manufacture cell and gene therapies. This week, Mark Battaglini, ARM's Chief Strategy Officer, joined Steve Favaloro, CEO of Genezen, at ARM Studios during #CGMesa24 to discuss how manufacturers are navigating these changes and embracing new opportunities.

Last month, Jimi Olaghere achieved a major milestone for the sickle cell community by reaching the summit of Mount Kilimanjaro. Rita Johnson-Greene, ARM's Chief Operating Officer, unpacks what this new milestone means to him and more during an ARM Studios interview this week at our 2024 Cell & Gene Meeting on the Mesa. Want to hear his whole story? If you're attending #CGMesa24, catch his fireside chat this morning at 9:00am AZ local time in the FLW Ballroom.