Highlights & Summary

Intro

July was an exciting month for the next-generation therapeutics space, full of new financing rounds, as well as clinical trial and regulatory updates.  

Happy reading!

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Cell Therapy

1. Umoja Biopharma Gets FDA Clearance of IND for UB-VV111, a CD19 CAR-T | Regulatory

2. Caribou Announces Lay Offs – Around 12% of Staff to be Affected | Personnel

3. Artiva Aims For $116M In Second Attempt At IPO to Boost Pipeline | Financing

4. Cartesian Raises $130M Along With Clearing An Autoimmune CAR-T Trial | Financing

5. J&J and Legend Biotech Announces Positive Overall Survival Results of Phase 3 CARTITUDE-4 Trial | Clinical Trial

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Gene Therapy  

1. Pfizer Receives EMA Approval for Durveqtix | Regulatory

2. Pfizer Announces Positive Results from Ph3 Hemophilia A Study | Clinical Trial

3. AskBio Receives Fast Track for Parkinson’s Asset | Regulatory

4. Beacon Therapeutics Raises $170M Series B | Financing

5. Croda Joines CGT Catapult Consortium to Advance AAV Development | Manufacturing

Oligo Therapy  

1. BioNTech Cancer Therapeutic Meets Primary Endpoint for Advanced Melanoma Drug Clinical Trial | Clinical Trial

2. Rona Therapeutics Secures $35M in Series A+ Financing Round | Financing

3. Sirnaomics Completes IND-enabling Studies for Novel siRNA Cardiovascular Disease Therapeutic  | Clinical Trial

4. AstraZeneca and Aptamer Group Partner to Create siRNA Therapeutics using Novel Targeted Delivery Platform | Partnership  

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Cell Therapy  

Umoja Biopharma Gets FDA Clearance of IND for UB-VV111, a CD19 CAR-T | Regulatory

Umoja Biopharma has received FDA approval to begin a Phase 1 trial of UB-VV111, its in vivo gene therapy for blood cancer, marking a potential shift from traditional ex vivo CAR-T therapies that require extensive cell manipulation. This new approach using the company's VivoVec platform aims to allow the body to generate its own CAR-T cells, reducing the complexity and cost associated with current therapies. The trial will target patients with relapsed or refractory large B cell lymphoma and chronic lymphocytic leukemia, as Umoja seeks to make CAR-T therapy more accessible and efficient.

Caribou Announces Lay Offs – Around 12% of Staff to be Affected | Personnel

Caribou Biosciences is reducing its workforce by 12%, eliminating 21 jobs to extend its cash runway and concentrate on its allogeneic CAR-T cell therapy platform. The company, co-founded by Jennifer Doudna, is discontinuing its CAR-NK research to focus on ongoing trials in B cell lymphoma and upcoming studies in autoimmune diseases and other cancers. This move follows Caribou's struggle with its stock value, which has dropped by 52% year-to-date. The layoffs will be completed by the end of September, leaving the company with approximately 145 employees.

Artiva Aims For $116M In Second Attempt At IPO to Boost Pipeline | Financing

Artiva Biotherapeutics is aiming to raise about $116 million from its upcoming IPO to expand its pipeline of off-the-shelf cell therapies for autoimmune and cancer treatments. The company plans to offer 8.7 million shares priced between $14 and $16. This move follows a delayed initial IPO attempt in 2021 and the recent termination of a partnership with Merck. As of the end of March, Artiva reported having $62 million, with significant investments in R&D. Major shareholders include GC LabCell, 5AM Ventures, venBio, and RA Capital. This IPO comes amid a challenging environment for biotech public offerings, despite a recent uptick in private capital inflows into the sector.

Cartesian Raises $130M Along With Clearing An Autoimmune CAR-T Trial | Financing

Cartesian Therapeutics reported that its lead mRNA cell therapy, Descartes-08, achieved its primary endpoint in a Phase 2b study for myasthenia gravis, showing significant improvement in 71% of patients compared to 25% in the placebo group. This announcement follows the company's recent public debut via a reverse merger with Selecta Biosciences. Cartesian is planning an end-of-Phase 2 meeting with U.S. regulators by year-end. The company is also exploring the therapy’s potential in systemic lupus erythematosus with a new Phase 2 study underway and has secured $130 million in funding to support its developments.

J&J and Legend Biotech Announces Positive Overall Survival Results of Phase 3 CARTITUDE-4 Trial | Clinical Trial

In a significant late-stage study, Johnson & Johnson and Legend Biotech's CAR-T therapy, Carvykti, demonstrated improved survival for multiple myeloma patients who had undergone one to three prior treatments, positioning the therapy earlier in treatment lines. The study, which led to Carvykti's expanded approval in April, compared its efficacy against standard therapies but did not release detailed survival data, promising more information at an upcoming medical conference. This development follows Carvykti's initial approval for patients who had exhausted most other options. With $500 million in revenue last year, Carvykti now surpasses sales of competing therapies like Abecma. J&J, in partnership with Novartis, is also working to double Carvykti’s production capacity by the end of 2024 to meet growing demand.

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Gene Therapy

Pfizer Receives EMA Approval for Durveqtix | Regulatory

Pfizer has received conditional marketing authorization from the European Commission for Durveqtix (fidanacogene elaparvovec), a gene therapy for moderate to severe hemophilia B in adults without factor IX inhibitors or detectable antibodies to AAV serotype Rh74. This authorization is based on results from the Phase 3 BENEGENE-2 study, which showed Durveqtix significantly reduced annualized bleeding rates compared to standard factor IX prophylaxis and was well-tolerated. The therapy was previously approved by the U.S. FDA under the name Beqvez.

Pfizer Announces Positive Results from Ph3 Hemophilia A Study | Clinical Trial

Pfizer has reported positive topline results from the Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for moderate to severe hemophilia A. The therapy achieved both non-inferiority and superiority in reducing the annualized bleeding rate compared to routine Factor VIII prophylaxis. Key secondary endpoints also demonstrated significant improvements, including a 98.3% reduction in treated annualized bleeding rate. The news comes at a time when BioMarin considers divesting its approved hemophilia A treatment, Roctavian due to sluggish sales.

AskBio Receives Fast Track for Parkinson’s Asset | Regulatory

Bayer and AskBio have announced that the U.S. FDA has granted Fast Track Designation to AB-1005, a gene therapy for moderate Parkinson’s disease. Additionally, AB-1005 has received the UK MHRA Innovation Passport for the same indication. AB-1005, an investigational AAV2 gene therapy, aims to treat Parkinson’s by delivering the glial cell line-derived neurotrophic factor (GDNF) directly to the brain. The FDA Fast Track and UK MHRA Innovation Passport designations are intended to accelerate development and regulatory review, with a Phase II clinical trial currently underway.

Beacon Therapeutics Raises $170M Series B | Financing

Beacon Therapeutics announced a $170 million Series B fundraise to advance its ophthalmic gene therapies for retinal diseases. Led by Forbion, the financing also included contributions from Syncona Limited, Oxford Science Enterprises, the University of Oxford, TCGX, and Advent Life Sciences. The funds will support the clinical development of AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and the Phase 1/2 trial for Dry Age-related Macular Degeneration (dAMD).  

Croda Joines CGT Catapult Consortium to Advance AAV Development | Manufacturing

The Cell and Gene Therapy Catapult (CGT Catapult) has formed a cross-industry consortium to enhance the understanding and optimization of AAV formulations for gene therapies. The consortium includes Croda, ikarovec, and UCL’s Department of Biochemical Engineering, aiming to address challenges related to the shelf-life and potency of AAV-based therapies. This collaboration will focus on improving AAV formulation, characterization, and stability, with the goal of advancing the development of AAV-based gene therapies.

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Oligo  

BioNTech Cancer Therapeutic Meets Primary Endpoint for Advanced Melanoma Drug Clinical Trial | Clinical Trial

mRNA therapeutic candidate BNT11 showed an improved response rate when used to treat patients with advanced melanoma in a Phase 2 trial. The inhibitor was used in combination with PD-1 checkpoint inhibitor cemiplimab. In earlier work, BNT111 was shown to activate tumor-antigen-specific T cells, boosting the immune system’s anti-tumor response.  

Rona Therapeutics Secures $35M in Series A+ Financing Round | Financing

Rona Therapeutics, known for developing next-generation RNA platforms utilizing siRNA, has secured $35M in its latest financing round spearheaded by LongRiver Investments. Contributions were included from Zhaode Investment, ZhongQi Capital, Lilly Asia Ventures and BioTrack Capital. Rona’s first product, RN0191, aimed at treating atherosclerotic cardiovascular disease, has recently completed Phase I clinical trials in Australia and China.  

Sirnaomics Completes IND-enabling Studies for Novel siRNA Cardiovascular Disease Therapeutic  | Clinical Trial

Sirnaomics finished IND-enabling studies for a cardiovascular disease therapeutic targeting apolipoprotein C3 (ApoC3) via siRNA. STP125G showed strong efficacy over 13 weeks and was tolerable in high doses in a non-human primate model. The data further validated STP125G as a therapeutic candidate for hypertriglyceridemia and other cardiovascular diseases.  

AstraZeneca and Aptamer Group Partner to Create siRNA Therapeutics using Novel Targeted Delivery Platform | Partnership

The partnership between AstraZeneca and Aptamer group will be leveraging Aptamer Group’s non-viral Optimer platform for selective siRNA delivery. The platform is being evaluated for the potential for licensing by AstraZeneca and has demonstrated efficacy in targeting hepatic stellate cells responsible for fibrotic liver disease in in vitro studies.