Gene Editing to Cross-Species Transplantation: The Most Compelling Healthcare Stories of 2022
Back Bay's team is fortunate to work behind the scenes in healthcare development and play a part in many unfolding stories, but we’re still inspired when we read stories from the intersection of good science and good business. Our morning dive into STAT, Endpoints, JAMA, etc. never ceases to bring fascinating stories of clinical and business feats to light.
Last year was a milestone year in terms of public health stories, from the profound and consequential reduction of access to women's healthcare across the United States to monkeypox and the ongoing complexities around the management of Covid-19, there are dozens of important themes emerging in healthcare to which we are all paying close attention.
Our purview lies specifically in healthcare development and the advancement of treatments that better human care.
In the development arena, the healthcare stories we track include news around important data readouts, partnerships, and pricing—especially around novel gene therapies—and regulatory concerns, such as The Inflation Reduction Act, Accelerated Approval Reform and others.
In our latest podcast, we cover three important healthcare development stories from 2022, including:
The progress in CRISPR and gene editing - Back Bay has been deeply involved in the evolving field of gene therapy and gene editing over the past years. Investor and clinical interest in this area is growing at a profound rate. Importantly,
On the ex vivo application front, Bluebird got an exciting win with FDA approval for Zynteglo in transfusion-dependent thalassemia. CRISPR Therapeutics and Vertex partnered on a couple of assets with promising initial data.
On the in vivo application front, the Editas asset Edit 101 showed mixed results in LCA 10 (an inherited form of blindness), but ultimately was scrapped. Intellia generated interesting data in transthyretin amyloidosis or ATTR amyloidosis, and this past year we also saw the entry of a base editor into a clinic.
Back Bay continues to monitor and advise how companies differentiate themselves in 2023 as the field of gene editing becomes more "crowded" and as we begin to understand more about the key attributes patients and physicians use to drive their decision-making process.
Forward momentum for gene therapies approvals and reimbursement - Last year brought positive momentum for gene therapies in later-stages, especially around regulatory approvals. There were four major approvals: three in the FDA and an additional one in Europe. Two were from Bluebird (Zynteglo and Skysona), as well as Hemgenix from CSL Behring and Roctavian approved in Europe.
Bluebird's Zynteglo launch in Europe (and their failure to ultimately cross the finish line in Germany and the UK) prompts questions on health technology assessment (HTA) agencies and country-specific criteria for pricing and reimbursement decision making.
Xenotransplantation – In early 2022, a cardiac team at the University of Maryland School of Medicine performed a xenotransplant, transplanting a pig heart into a 57-year-old male patient who had terminal stage heart failure, an incredible scientific milestone.
Thanks to our resident healthcare news experts who poured through hundreds of articles to bring you this overview, Brendan Wang, Christian Thienel and Peter Bak, and with special thanks to Micheka Fenelon.
Want to contribute to this roundup? Be in touch with our healthcare advisors.