Highlights & Summary
Intro
April was a busy month for the next-generation therapeutics field, full of manufacturing news and financing deals. Other key announcements include a number of new partnerships and clinical trial updates.
Happy reading!
Cell Therapy
- CTRL Therapeutics Raises $10M Seed Financing to Advance Cell Therapy Platform for Solid Tumors | Financing
- VintaBio Emerges from Stealth Mode with $64M Backing to Help Alleviate the Viral Vector Bottleneck in Cell and Gene Therapy Development | Manufacturing
- BMS Strengthens Cell Therapy Capabilities by Adding New U.S. Manufacturing Facility for Viral Vector Production | Manufacturing
Gene Therapy
- Complement Therapeutics Raises €72M Series A | Financing
- Sangamo Makes Layoffs as Pipeline is Pared Back | Personnel
Oligo Therapy
- FDA Grants Accelerated Approval for SOD1-ALS | Regulatory
- Moderna and Merck Announce mRNA-4157 in Combination with KEYTRUDA Demonstrated Superior Recurrence-Free Survival | Clinical Trial
- Moderna Establishes Agreement with Government of the Republic of Kenya for mRNA Manufacturing | Manufacturing
Antibody-Drug Conjugates
- BioNTech and DualityBio Announced an Exclusive ~$1.6B Global Licensing Agreement For 2 Investigational ADCs in Oncology | Partnership
- Abdera Therapeutics Debuts With $142 Million in Financing to Advance Antibody-Based Radiopharmaceuticals | Company Creation
- Tubulis and BMS Announce a $1B Licensing Agreement For The Development of Next-Gen ADCs | Partnership
- Mythic Therapeutics Announces First Non-Small Lung Cancer Patient Dosed With MYTX-011 | Clinical Trial
- Innate Pharma Enters a ~$415M Licensing Agreement with Takeda For The Development of ADCs For Celiac Disease | Partnership
- Abbvie Ended The Development of Its Anti-TNF Lead ADC Asset, ABBV-154, Due To Doubts About Its Competitiveness | Clinical Trial
Cell Therapy
CTRL Therapeutics Raises $10M Seed Financing to Advance Cell Therapy Platform for Solid Tumors | Financing
CTRL Therapeutics will be using the funding to make further progress on their platform focused on extracting circulating tumor-reactive lymphocytes (cTRLs) from blood which builds on the advantages of TIL therapies while providing greater efficacy by improving the immune-phenotype, quality and consistency of therapeutic cells harvested.
Gamida Cell’s Allogeneic Cell Therapy Omisirge Receives FDA Approval | Regulatory
Omisirge will be used to help reduce the risk of infection in patients with hematologic malignancies who receive umbilical cord blood transplantation by improving the neutrophil recovery rate. The approval comes as a much needed break for Gamida Cell, as the beleaguered biotech saw its funding dry up prompting two waves of lay-offs during its Omisirge development and approval journey.
Bill & Melinda Gates Foundation Invest $5M in Smart Immune | Financing
Smart Immune is developing ProTcell, a proprietary, thymus-empowered T-cell therapy platform to treat cancer and infectious disease. The funding will mainly be used in further progressing Smart Immune’s lead asset, SMART 101, an allogeneic cell therapy, through Phase I/II in acute leukemia patients.
VintaBio Emerges from Stealth Mode with $64M Backing to Help Alleviate the Viral Vector Bottleneck in Cell and Gene Therapy Development | Manufacturing
VintaBio was co-founded by Junwei Sun and Dr. Shangzhen Zhou, who developed the first viral vectors used for life-saving therapies. Together with David Radspinner, who previously held positions at Cytiva and ThermoFisher, as CEO, VintaBIo has a very experienced team at its helm. The company just opened its custom-built 22,500-square-foot Philadelphia manufacturing facility that is now open and accepting orders.
BMS Strengthens Cell Therapy Capabilities by Adding New U.S. Manufacturing Facility for Viral Vector Production | Manufacturing
Manufacturing is clearly top of mind in the cell therapy space, with BMS being another player in this space that focused on expanding their capabilities in April. BMS expanded capabilities to enable in-house viral vector production through a facility in Libertyville, Illinois, following the company’s execution of an agreement with Novartis. The facility will be able to support both BMS’ CAR T cell therapies.
Gene Therapy
Ginkgo Bioworks Acquires StrideBio’s Capsid Discovery Platform | M&A
Ginkgo Bioworks acquired the AAV discovery engine of StrideBio to bolster the former’s viral discovery and development services. Gingo in recent years has made several acquisitions to build out their discovery service capabilities across a range of next-generation modalities. The firm plans to license out or sell off Stride’s discrete therapeutic programs.
Complement Therapeutics Raises €72M Series A | Financing
Despite a tough macroeconomic climate, Complement Therapeutics, a German gene therapy company raised €72 million Series A. The funds will help support continued clinical development of their lead asset for geographic atrophy (GA) secondary to dry-AMD. The FDA recently approved the first treatment for GA that similarly targets the complement pathway, which the company sees as validation for their strategy.
New Study Elucidates Process of Viral Particle Assembly | Research
New work published in Nature elucidates the formation and packaging of viral genomes in their shell. The work found that the capsid forms around the viral genome through phase separation. The study identified a protein on the virus that creates the viral condensates within the cell, which can then be fully assembled.
Sangamo Makes Layoffs as Pipeline is Pared Back | Personnel
Having recently lost two large-pharma partners, Sangamo is paring back its workforce and doubling down on its late-stage asset for Fabry disease. The company is planning to shift its core R&D focus generally to neuropathic pain and other associated CNS disorders. The company recently disclosed data on their Fabry asset from their Phase 1/2 study, which showed a good safety profile and elevated expression of alpha-galactosidase A, which provides hope for an effective treatment.
REGENXBIO Receives Fast-Track for DMD Candidate RGX-202 | Regulatory
REGENXBIO received FDA Fast-Track designation for their asset for treating Duchenne Muscular Dystrophy (DMD). Presently in Phase 1/2 testing, the asset is competing in a crowded space, presently lead by Sarepta’s SRP-9001, who’s BLA was filed late last year. The agency originally said no Adcom would be needed for SRP-9001, but then reversed course.
Oligo
FDA Grants Accelerated Approval for SOD1-ALS | Regulatory
Biogen Inc announced the FDA approval of QALSODY, an antisense oligonucleotide that binds to SOD1 mRNA and treats amyotrophic lateral sclerosis (ALS) who have the superoxide dismutase 1 (SOD1) mutation. Participants had less decline from baseline compared to the placebo and experienced a 55% reduction in Nfl plasma levels.
Moderna and Merck Announce mRNA-4157 in Combination with KEYTRUDA Demonstrated Superior Recurrence-Free Survival | Clinical Trial
Moderna and Merck announced the results from the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial evaluating mRNA-41567 in combination with KEYTRUDA for patients with high-risk melanoma. The study demonstrated a meaningful improvement in recurrence-free survival and risk of death.
Moderna Establishes Agreement with Government of the Republic of Kenya for mRNA Manufacturing | Manufacturing
Moderna, in partnership with the Government of the Republic of Kenya, will build an mRNA facility that will have the capacity to produce up to 500 million vaccine doses annually. Moderna has committed to establish mRNA manufacturing facilities across the globe in an effort to bridge health security and public health.
Sensible Raises $4.2M in Pre-Seed Financing for mRNA Manufacturing | Financing
Sensible Biotechnologies, Inc., is an mRNA platform company and recently reported $4.2M in pre-seed funding. Sensible has a cell-based platform for manufacturing mRNA that has the potential to improve scalability and cost-efficacy.
ADCs
BioNTech and DualityBio Announced an Exclusive ~$1.6B Global Licensing Agreement For 2 Investigational ADCs in Oncology | Partnership
BioNTech is expanding its ADC pipeline and deepening its presence in oncology by acquiring licensing rights for 2 DualityBio assets, outside of China. DB-1303 is an anti-HER2 ADC that is currently in phase 2 of development, and has shown efficacy in both HER2-positive and HER2-low tumors. DB-1311 is currently in the preclinical stage. Both assets are conjugated to a topoisomerase-1 inhibitor payload.
Abdera Therapeutics Debuts With $142 Million in Financing to Advance Antibody-Based Radiopharmaceuticals | Company Creation
Abdera, which has five ongoing programs, has raised $142 million in a combined Series A and B financing round to develop tunable precision radiopharmaceuticals for cancer using its advanced antibody engineering ROVEr platform. The company's primary asset, ABD-147, specifically targets DLL3, which is upregulated in small-cell lung cancer (SCLC). Abdera intends to submit an IND for ABD-147 in 2024.
Tubulis and BMS Announce a ~$1B Licensing Agreement For The Development of Next-Gen ADCs | Partnership
BMS will receive access to Tubulis' Tubutecan payloads and P5 conjugation platform, and will be responsible for developing and marketing the resulting ADC assets. Tubulis' platform enhances ADC stability, which leads to an increase in on-target payload delivery and a reduction in off-target toxicity. ADCs have become one of the cornerstones of BMS's oncology arsenal, with this agreement following a $3.1B partnership with Eisai in 2021 and its acquisition of Turning Point Therapeutics for $4.1B in 2022.
Mythic Therapeutics Announces First Non-Small Lung Cancer Patient Dosed With MYTX-011 | Clinical Trial
Mythic Therapeutics has announced that the first patient has been administered MYTX-011 in the KisMET-01 phase 1 trial, which is assessing its efficacy in treating NSCLC. MYTX-011 is an anti-cMET ADC that is conjugated to the MMAE payload. In addition, the company presented preclinical data for its primary asset at AACR, demonstrating increased effectiveness in NSCLC mouse and monkey models, as well as ex-vivo data across 60+ cancer cell lines, in contrast to other anti-cMET ADCs in development. DeciBio's TheraTrack reveals that currently, six companies are each working on at least one anti-cMET ADC.
Innate Pharma Enters a ~$415M Licensing Agreement with Takeda For The Development of ADCs For Celiac Disease | Partnership
The two companies have entered into an exclusive agreement to carry out research and development on ADCs that target an undisclosed target in Celiac Disease. Takeda will receive global rights to use a selected set of Innate Pharma antibodies to create, manufacture, and market the resulting ADCs. DeciBio's Theratrack reports that there are currently over 40 active non-oncology ADC programs in various stages of development.
Abbvie Ended The Development of Its Anti-TNF Lead ADC Asset, ABBV-154, Due To Doubts About Its Competitiveness | Clinical Trial
ABBV-154 was under study for Rheumatoid Arthritis (RA), Crohn's disease (CD), and Polymyalgia Rheumatica (PMR). Although the asset performed as anticipated, it only achieved the expected results at the highest dose, causing alterations in biomarkers consistent with systemic steroid exposure. AbbVie concluded that the asset would not have a competitive safety profile in comparison to commercially available products and other assets in development. Nevertheless, the company is still pursuing RA using a different ADC from its portfolio, ABBV-3373.
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