Highlights & Summary
Welcome to the inaugural edition of DeciBio’s Next-Generation Therapeutics Newsletter!
Cellular, oligonucleotide, and AAV-based therapeutics are at the forefront of precision medicine, and we’re here to keep you up to date on important developments across these modalities. Each month we will curate a list of recent, impactful events drawn from research reports, clinical trial news, and corporate updates, and share them along with expert insights and commentary.
This month we have seen major new trial developments and partnership changes in the cell therapy space, creative funding rounds across gene therapies, and significant manufacturing expansions for oligonucleotide therapies.
Happy reading!
Cell Therapy
- Caribou Biosciences reported impressive human data for its allogeneic off-the-shelf CAR-T therapy | Clinical Trials
Gene Therapy
- FDA lifts clinical hold on Pfizer’s gene therapy for hemophilia A | Clinical Trials
- Phase 1 trial of gene therapy delivery of broadly neutralizing antibodies in HIV+ adults | Clinical Trials
Oligo Therapy
- Eli Lilly plans to invest $2.1 billion in new next generation manufacturing sites in Indiana | Investment
Cell Therapy
- Caribou Biosciences Reported impressive human data for its allogeneic off-the-shelf CAR-T therapy | Clinical Trials
The CB-010 product demonstrated a 100% overall response rate and 80% complete response rate in relapsed/refractory non-Hodgkin's lymphoma. Caribou’s CRISPR gene-editing strategy and further allogeneic cell therapy development are both worth watching. Expect further updates next month at the European Hematology Association Congress on June 10th.
Following a clinical hold (after a patient death) on the trial for the ATA2271 autologous CAR-T targeting mesothelin, Bayer has canceled a partnership with Atara Biotherapeutics. The move is a major shift for Bayer’s CAR-T plans and a big hit to Atara, who hopes to continue the trial.
The US Food and Drug Administration granted approval for the use of Kymriah (tisagenlecleucel), a CD19-directed CAR-T cell therapy, in adult patients with relapsed or refractory follicular lymphoma. This approval was granted based on data from the Phase II ELARA trial (NCT03568461), in which Kymriah demonstrated an 86% overall response rate and 68% complete response rate. Kymriah, the first CAR-T cell therapy approved in the US, is now approved for use in three indications across both pediatric and adult populations.
Gene Therapy
- FDA lifts clinical hold on Pfizer’s gene therapy for hemophilia A | Clinical Trials
The company is still maintaining a voluntary pause after earlier findings of potential increased risk of blood clots. This program is chasing Biomarin’s valrox, which similarly faced another snag with the FDA as the agency recently requested “additional analysis” to be included in Biomarin’s BLA.
- Phase 1 trial of gene therapy delivery of broadly neutralizing antibodies in HIV+ adults | Clinical Trials
Early proof-of-concept clinical study demonstrating the ability of an AAV vector to code for neutralizing antibodies against HIV. This could open the door to new indications for AAV beyond correction of monogenic disorders
SwanBio, which recently had to layoff a quarter of its staff, announced a new round of financing of $56M composed of only current insiders to help support the start of their Phase I/II trial for adrenomyeloneuropathy
Oligo Therapy
Verve Therapeutics has received approval to start clinical testing of its VERVE-101 clinical trial program in New Zealand, to address familial hypercholesterolemia using CRISPR technology, with the first human phase 1 data expected in 2023. Following Intellia Therapeutics (NTLA-2002) and Editas Medicine (EDIT-101), Verve Therapeutics’s asset is now the third in-vivo genome editing treatment to enter human trials.
- Eli Lilly plans to invest $2.1 billion in new next generation manufacturing sites in Indiana | Investment
Eli Lilly unveiled its $2.1 billion investment plan to build two new manufacturing facilities in central Indiana. The plants will be designed to boost Lilly’s next generation manufacturing capacity for active ingredients and new genetic medicine therapeutics, highlighting its dedication to strengthening its genetic medicine portfolio.
Following positive results announced in April 2022 for the ARCT-154 self-amplifying mRNA COVID-19 vaccine, Arcturus Therapeutics reported strong 3-month durability results from the ongoing phase 1/2 booster trial. The company has already initiated plans for international phase 3 studies.
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