Highlights & Summary
August was a month that had some significant new therapeutic approvals as well as company launches, in addition to several financing rounds and partnerships. Read below to find out more about these new key developments.
- Bluebird’s Zynteglo approval moves the needle on CD34-based therapies and beta-thalassemia | FDA Approval
- ElevateBio moves to commercialize work from their ongoing academic collaboration by starting a new company | Company Creation
- Affini-T makes a strong hire for its TCR-T pipeline with ex-Amgen Dirk Nagorsen coming on as CMO | Personnel
- Voyager Therapeutics Set to Re-Invent Itself Under Sandrock’s Leadership with Focus on CNS | Research
- Versant launches another one, this time for prevision gene editing | Company Creation
- FDA Grants Orphan Drug Designation to ABX1100, an investigational Centryin-siRNA, for Treatment of Pompe Disease | Clinical Approval
- Eleven Therapeutics, an siRNA and AI biotechnology company has raised 22 million seed funding | Financing
- Cure Rare Diseases receives FDA approval for a Duchenne muscular dystrophy Crispr therapy clinical trial | Clinical Trial
- Novel oligo-delivery platform from Dyne Therapeutics efficiently restores dystrophin in mdx mine | Pre-Clinical
Zynteglo paves the way as a potentially curative for beta-thalassemia and a future for cell-based gene edited CD34 therapies. This comes as a win for both Bluebird and gene edited therapies as Bluebird has faced a number of obstacles to this point.
Cell therapy manufacturing activity heats up as companies expand internal capabilities. Cell therapy CMC remains a limitation and continues to be a hotspot for further development.
ElevateBio has had an ongoing collaboration with Boston Children’s Hospital and the George Daley Lab. This collaborative effort will continue to develop iPSC-based allo cell therapies.
Dirk Nagorsen comes with extensive expertise with approved Kras therapy and T-cell engager therapy. Affini-T is focusing efforts on Kras and T-cell engager therapies.
Celyad will continue their Phase 1b clinical trial after FDA lifts hold | Clinical Trials
With 2 fatalities in the study, Celyad will continue trials with allo therapies with novel gene edits (NKG2D and TIM - a proprietary technology). Allo Car-T is a hot space in the market as companies push to bring therapies to market.
This model is common in order to build out internal manufacturing capabilities. This serves two purposes as either a valuable asset or as collateral. The appetite for “curative” gene therapies remains strong.
This new partnership will focus on both cardiac and neuromuscular indications. In turn, this will strengthen Sarepta’s current pipeline focused on muscular dystrophies.
New opportunities are on the horizon despite the company facing setbacks in prior pipelines focused on Parkinson’s and Huntington’s disease.
New in-vivo editing technology could simplify the delivery of base editors and other editing machinery in vivo.
Versant launches another one, this time for prevision gene editing | Company Creation
Versant is focusing on delivering large genetic payloads with a non-viral platform to reduce immunogenicity, accomplishing two goals - increasing payloads and reducing immune response, with gene editing. This launch comes on top of previous launches that Versant made from its Ridgeline Discovery Engine in Basel.
Aro Biotherapeutics, a Philadelphia based biotechnology company has received Orphan Drug Designation from the FDA for a Centryin-siRNA conjugate that targets Gys1, a gene in muscle that is involved in muscle glycogen synthase. Pompe Disease is a genetic disease that results in progressive and debilitating muscle weakness.
Orna Therapeutics closed $221 million Series B financing for developing circular RNA and lipid nanoparticle delivery.
With 22 million in seed funding raised, Eleven Therapeutics continues to develop siRNAs leveraging its AI capabilities. Investors include Bill & Melinda Gates Foundation among others.
FDA approves Cure Rare Disease to administer a novel Crispr therapeutic that targets exon 1 mutations on the dystrophin gene. The trial will occur at University of Massachusetts Medical School with the goal of stabilizing progression of DMD.
Using the FORCE platform, with only one dose of the curative therapy, dystrophin expression was significantly increased in heart and skeletal muscle. While this study is pre-clinical, this presents an exciting finding for oligo-based therapies in DMD.