Last month, Alliance for Regenerative Medicine released its latest Sector Snapshot report, highlighting the impressive growth and development of the sector over the past year.
The report highlights the record-breaking number of regulatory approvals of gene therapies for rare diseases in 2022. Despite a challenging financing environment, the sector is poised to continue to make significant progress in 2023.
CRISPR is the trend to monitor, with Vertex Pharmaceuticals and CRISPR Therapeutics looking to secure the first-ever approval of a CRISPR gene-editing therapy.
In order to realize the potential of these life-changing therapies, healthcare systems need to be prepared to deliver them to patients.
Here are some of the key takeaways from the report.
What stood out in 2022?
- First ever ‘Off-the-shelf’ T-Cell therapy approved globally
- 3 CAR-T Cell therapies expanded for treating different cancers; 2 approved as earlier-line treatments
- 3 new gene therapies approved for rare diseases (a new record)
- Investment of $12.6B across 478 deals
- 2,200 clinical trials
Trend to watch: CRISPR
Exacel, the first CRISPR therapy for treating sickle cell disease, is up for approval this year.
This represents a significantly larger patient population than diseases targeted by previous approved gene therapies.
CRISPR has overtaken other gene editing technologies (such as TALEN and ZFN) in clinical trail and is being tested in various disease areas.
Advanced forms of gene editing such as prime editing and epigenetic editing are emerging to improve accuracy and efficiency.
What can we anticipate for the rest 2023?
The next wave of CGTs is arriving fast, with decisions expected on:
- 10 CGTs in the US, including gene therapies for rare diseases
- First-ever CRISPR gene editing therapy
- First adoptive cell therapy for a solid tumor
- First gene therapy for Duchenne muscular dystrophy
Aliance for Regenerative Medicine full article: Regenerative Medicine: Disrupting the Status Quo
