What's Next For Advanced Therapies?

The advanced therapies sector is growing exponentially. The Alliance for Regenerative Medicine (ARM) reports that the sector raised $23.1 billion in 2021 – a 16% year-over-year increase. That figure includes $1.9 billion in private financing and $9.8 billion in venture funding.¹

This paper is based on a panel discussion, sponsored by Precision ADVANCE, on the future of advanced therapies, featuring the following industry leaders:

• Anshul Mangal, discussion moderator; President, Project Farma (PF) and Precision ADVANCE: Under Mangal’s leadership, PF pioneered the industrialization of advanced therapies and created the Advanced Therapy Manufacturing Playbook, which has helped develop the world’s largest cell and gene therapy manufacturing footprint. PF was acquired by Precision Medicine Group in 2020 to be the cornerstone of Precision ADVANCE, a collection of interconnected services and complementary teams uniquely focused on the complexities of clinical, regulatory, manufacturing, and commercial needs to bring a cell or gene therapy to market.
• R.A. Session, II, President, Founder, and CEO, Taysha Gene Therapies: With a focus on monogenic diseases of the central nervous system, Taysha has a portfolio of 26 programs (including 4 in the clinic) across the company’s neurodegenerative, neurodevelopmental, and genetic epilepsy franchises. All programs incorporate adeno-associated virus serotype 9 vectors, intrathecal delivery, and HEK 293 suspension cells, which are commonly used for protein and virus production.
• Palani Palaniappan, Executive Vice President and Chief Technology Officer, Aruvant Sciences: Aruvant’s lead product candidate is an autologous cell therapy for sickle cell disease (SCD), with a pivotal trial scheduled to launch in 2022. Aruvant also seeks to expand its pipeline to address metabolic diseases and other disease areas such as hypophosphatasia and spinal muscular atrophy (SMA).
• Derrell Porter, Founder and CEO, Cellevolve Bio: A development and commercialization platform company focused on cell therapies, Cellevolve is emerging from quiet mode, signing up partners and recruiting team members and advisors. In the summer of 2021, Cellevolve signed its first collaboration for a cell therapy for progressive multifocal leukoencephalopathy (PML), which is set to enter pivotal trials in early 2022.
• Claudia Zylberberg, Founder and Executive Chair, Akron Bio: For the last 17 years, Akron has been working on the “intel inside” to create a standardized industrial platform for cell therapies to ensure product batch consistency and manufacturing at scale, while reducing cost of goods and meeting regulatory standards. Akron recently opened a new facility to produce plasmid DNA (pDNA), a supportive biomanufacturing infrastructure for creating a “plug and play” platform for customers.
• Phil Cyr, Senior Vice President, Customer Solutions, Precision Value & Health: Precision aims to break down silos and enhance communication across the spectrum of cell and gene therapy development, including specialty laboratories, clinical trials, manufacturing, health economics and outcomes research, marketing, payer relations, and patient engagement. By demonstrating clinical and economic value to payers based on data and manufacturing capabilities, Precision helps clients speed up the process to regulatory approval and reduce barriers to patient access.