5 Top Takeaways from ASGCT’S Q2 2022 Quarterly Data Report

Ryan Leahy
25 July 2022
CAR-X
Cell Therapy
Collaborations, Mergers & Acquisitions
Finance & Investment
Gene Therapy
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Phacilitate’s Ryan Leahy summarizes his top 5 takeaways from ASGCT’s Gene, Cell, & RNA Therapy Landscape: Q2 2022 Report, exploring the progress of clinical and commercial development in these advanced therapeutic areas this quarter.
Prevailing Patterns

The number of gene therapies in pre-clinical to pre-registration development increased from Q1 by 4% to 2024, 8 of which are in pre-registration stages.

A proportion of 73% of all the investigational gene therapies use ex vivo genetic modification – the same as the previous 2 quarters.

The number of anti-cancer gene therapies in development has overtaken those targeting rare diseases, with 1089 and 1003 under investigation, respectively. Of those targeting rare diseases, 8 out of 10 are for oncological indications.

Similarly, looking at non-genetically modified cell therapy pipelines, the number of products in development as anticancer drugs exceeds those for rare diseases at 292 and 263, respectively. However, 63% of these rare diseases focus on non-oncological disorders – with COVID-19 complications the main target.

Q2 saw a decrease of 20% in the number of gene therapy clinical trials compared to Q1.

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Note-Worthy start-ups

Q2 2022 saw an increase in volume and value of combined seed/Series A financing, with 17 start-ups raising $793.3 million, following two previous flat quarters.

  1. Tessa Therapeutics secured a $126m Series A financing, led by Polaris Partners, for its development of allogeneic CAR-T therapies targeting CD30-positive cancers.
  2. Satellite Bio reported $110m combined seed and Series A financing, led by aMoon Growth. This biotherapeutic company has an adaptive tissue platform to convert any cell type into bioengineered, allogeneic and implantable tissue.
  3. Code-bio had a successful $75m Series A financing, led by Northpond Ventures, as they utilize their non-viral 3DNA delivery platform for gene therapies to treat Duchenne muscular dystrophy and type 1 diabetes.
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RNA Pipeline on the Up

Q2 saw an increase in messenger RNA, RNA interference, and Oligonucleotides therapies – the RNA pipeline landscape growing 4% since 2022 Q1.

18 RNA therapies are currently approved, with 2 approvals of RNA-based therapies taking place in Q2. Gennova Biopharmaceuticals saw its COVID-19 vaccine for coronavirus approved in India, and Alnylam had its product Amvuttra approved in the U.S. to treat hereditary amyloidosis.

Development in mRNA, RNAi and antisense therapies continues to be dominated by pre-clinical investigations. 854 RNA therapies are currently in pre-clinical to pre-registration stages. Rare diseases are the indication area targeted by these therapies. Of these indications, Duchenne’s muscular dystrophy is the target for the highest number of investigational therapies at 28, whilst, pancreatic cancer is targeted by the highest number of therapies for an oncological disorder.

CAR-T Continues its Reign

CAR-T cell therapies continue their attack on oncological indications, accounting for all of the Q2 gene therapy approvals (including gene-modified cell therapies).

  • Novartis’ Kymriah, the OG CAR-T, added follicular lymphoma to its list of cancers it has proved effective against, gaining approval in this new indication across all of the countries it is available.
  • The EU and UK joined Q1’s U.S. approval of Legend Biotech’s CAR-T offering, Carvykti, for the treatment of multiple myeloma.
  • Another CAR-T to gain added locational approval was Celgene’s Breyanzi, now approved for patient treatment of diffuse large B-cell lymphoma and follicular lymphoma in the EU, UK and Canada.

CAR-T was also the most common therapy in the pipeline of genetically modified therapies representing 48%. Similarly, to the previous three quarters, 98% of CAR-Ts are in development against cancer.

 

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Who’s Acquiring Who?

9 acquisitions took place in Q2, up slightly from Q1. The most notable is Galapagos’ acquisition of CellPoint for $237 million, moving Galapagos into the thriving CAR-T market.

Other deals to shout about are GC Corp’s acquisition of CDMO BioCentriq for $73m, CureVac’s acquisition of Frame Theraputics for 34m, and, Evotec’s acquisition of Rigenerand for $25m.

However, total deal volume dropped 20% from Q1.

As cell, gene and RNA therapeutics continue to expand into new products, technologies, and disease areas, we are likely to see further advancement of pre-clinical and clinical developments in the next quarter, alongside some new and exciting acquisitions and start-up news.


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