2023 Cell and Gene Therapy Predictions
We surveyed 35 stakeholders across the Cell and Gene Therapy industry on what lies ahead in 2023.
Highlights & Summary
Intro
The field of Cell and Gene Therapy was off to a busy start in 2023, with 8 partnerships and acquisitions, in addition to notable clinical trial updates.
Happy reading!
Cell Therapy
- Fate Therapeutics terminates collaboration with Janssen to focus on other parts of its portfolio | Partnership
- ArsenalBio announces dosing of first patient with AB-1015 in its phase 1 trial for ovarian cancer | Clinical Trial
Gene Therapy
- FDA Lifts Hold on Astellas’ Candidate for Pompe Disease | Clinical Trial
- Homology Medicines Announces Updated Data on Clinical Assets | Clinical Trial
Oligo Therapy
- Moderna Acquires Oriciro Genomics | Acquisition
- Esperovax and Ginkgo Bioworks Announce Partnership | Partnership
- Metagenomi Closes Series B Financing with $275M | Financing
Antibody-Drug Conjugates
- First made-in-Singapore ADC approved by the FDA for a phase 1 trial | Clinical Trial
- Magenta Therapeutics pauses MGTA-117 phase 1/2 trial in R/R AML due to safety concerns | Clinical Trial
- Sunaffix enters $2 billion license agreement with Amgen | Partnership
Cell Therapy
Tessa to prioritize allogeneic CAR-T cell therapy platform | Commercial
Tessa announced that the company will prioritize development of its allogeneic “off-the-shelf” platform, while seeking strategic options for the development of its TT11 autologous CAR-T platform with other companies. The decision to prioritize comes after strong safety and efficacy data presented at ASH
Fate Therapeutics terminates collaboration with Janssen to focus on other parts of its portfolio | Partnership
After reviewing its portfolio of its natural killer (NK) cell products, Fate has decided to focus on advancing its most innovative and differentiated programs, and terminate its partnership with Janssen. Fate expects to have sufficient financial resources through the end of 2025 to capitalize on its iPSC-derived chimeric antigen receptor (CAR) NK and CAR T-cell programs. In total Fate received around $13M in milestone payments from the partnership.
ArsenalBio announces dosing of first patient with AB-1015 in its phase 1 trial for ovarian cancer | Clinical Trial
AB-1015 is ArsenalBio’s first internally discovered T cell medicine to enter clinical development and uses synthetic DNA programming to overcome tumor defense. The results of this trial will be used to validate ArsenalBio’s Integrated Circuit T (ICT) cell technology in humans, marking a significant milestone for the company.
Editas announced that it entered into an agreement where Shoreline can license Editas Medicine’s proprietary SLEEK (SeLection by Essential-gene Exon Knock-in) and AsCas12a gene editing technologies in addition to acquiring Editas Medicine’s preclinical gene edited induced pluripotent stem cell (iPSC) derived natural killer cell (iNK) programs and related manufacturing technologies.
Takeda-backed Ensoma buys a CRISPR upstart in addition to raising $85M | Acquisition
Ensoma had a busy month - it made an all-stock acquisition of Danish CRISPR upstart Twelve Bio, in addition to raising $85 million in its Series B. The Boston biotech will use the proceeds to bring its lead oncology program into the clinic, with the goal of targeting hematopoietic stem cells with a one-time, off-the-shelf therapy.
Gene Therapy
Capsida Biotherapeutics Announces Partnership with Eli Lilly | Partnership
Capsida announced a partnership with Eli Lilly’s subsidiary, Prevail Therapeutics. The goal is to leverage Capsida-designed capsids combined with Prevail’s cargo to address diseases of the central nervous system. The partnership came with $55M upfront and $685M in milestones. This expands the list of partners for Capsida, who previously signed agreements with AbbVie and CRISPR Therapeutics.
FDA Lifts Hold on Astellas’ Candidate for Pompe Disease | Clinical Trial
Astellas resolved the clinical hold on AT845, allowing for their FORTIS Phase I/II trial to continue. The hold was first placed in June of last year due to the discovery of nerve damage in a participant. This program marks one of only two programs still in development from the Audentes acquisition.
Editas Pares Back Pipeline Addressing Inherited Retinal Diseases | Pipeline
Editas announced a total company reprioritization, ending internal investment in EDIT-101 and EDIT-103, for Leber Congenital Amaurosis and Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa, respectively. Using AAV5 vectors, these programs were some of the highlights of the pipeline only a year ago, with EDIT-101 in clinical testing. The therapy utilized an AAV vector to deliver CRISPR machinery and a guide RNA to cut out the defective version of the CEP290 gene. The company will look for external partners to potentially advance these programs further.
Homology Medicines Announces Updated Data on Clinical Assets | Clinical Trial
Homology Medicines announced updates for HMI-103, their Phase I candidate to treat phenylketonuria (PKU). The therapy leverages homologous recombination to insert a liver-specific promoter to correct the defective PAH gene. The trial should readout data this year. There are at least 4 programs for PKU in development. Biomarin’s BMN-307 in Phase I/II testing was placed on hold in 2021, which is yet to be resolved. Other groups, such as Sangamo, have deprioritized their programs.
Oligo
Moderna Acquires OriCiro Genomics | Acquisition
Moderna acquires OriCiro Genomics for $85 million. OriCiro has a number of synthetic biology and enzyme technologies for cell-free DNA synthesis and amplification. OriCiro’s technology could be critical in improving mRNA manufacturing for Moderna.
AbbVie and Anima Biotech Announce Collaboration for mRNA Modulators | Partnership
AbbVie and Anima Biotech announced a collaboration to leverage Anima’s Lightning platform and AbbVie’s oncology experience. Anima’s Lightning platform is a novel approach for small molecular mRNA drug discovery. Anima will receive $42 million with additional opportunities for royalties.
Esperovax and Ginkgo Bioworks Announce Partnership | Partnership
Esperovax and Ginkgo are partnering to develop circular RNAs (circRNAs) for cancer therapeutics. This partnership will leverage Esperovax’s oral mRNA biologics and Ginkgo’s platform for cell programming. CircRNAs may have improved stability compared to linear mRNA.
Metagenomi Closes Series B Financing with $275M | Financing
Metagenomi brings in another $100M to close their Series B with $275M. Metagenomic is a genetic medicine company that has a number of next-generation gene editing systems. Key investors in this series B extension financing include Bayer and Moderna. Metagenomi plans to advance som eo their preclinical programs into clinical proof-of-concept with the additional financing.
ADCs
First made-in-Singapore ADC approved by the FDA for a phase 1 trial | Clinical Trial
EBC-129 targets the glycosylated form of CEACAM5/6, overexpressed in cancer cells, and is conjugated to the payload MMAE. CEACAM6 has been historically hard to target due to its normal expression on healthy cells. A*STAR is also working on developing a CDx IHC test for patient selection. According to DeciBio’s TheraTrack, one other ADC is in development targeting both CEACAM5/6 and is currently in preclinical development.
AstraZeneca to discontinue LUMOXITI from the U.S. Market in July 2023 | Commercial
Due to the low clinical uptake since approval and to the competitive landscape, AstraZeneca has decided to permanently withdraw the asset from the U.S. Market. LUMOXITI is a CD22-targeting ADC approved in 2018 for the treatment of R/R hairy cell leukemia.
Magenta Therapeutics pauses MGTA-117 phase 1/2 trial in R/R AML due to safety concerns | Clinical Trial
The company announced that it has voluntarily paused the clinical trial for its conditioning asset in R/R AML and MDS, due to a patient’s death following a grade 5 adverse event (respiratory failure and cardiac arrest). MGTA-117 is a CD117-targeting antibody conjugated to Amanitin with a DAR of 2.
Sunaffix enters $2 billion license agreement with Amgen | Partnership
Synaffix will be providing Amgen with access to its antibody conjugation platforms (e.g., GlycoConnect™, toxSYN™) for one ADC program with the option to expand the agreement for four additional programs. The deal consists of milestone payments, tiered royalties on sales etc., amounting to a potential $2B deal.
Samsung Biologics plans to enter the ADC Manufacturing space in early 2024 | Manufacturing
At JPM, the company revelaved its plan to enter the ADC manufacturing space and is planning to build an ADC manufacturing site and start production by early 2024. Samsung Biologics aims to become a global top-tier biopharmaceutical company in the space, by 2023.
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