Q1 2024 CGT Sector Sees 28 Strategic Collaborations, Led by Novartis-Voyager’s $1.3B Gene Therapy Deal

Mar 31, 2024 07:01 CST Updated 07:01

GSK

Pharmaceutical R&D Manufacturer

Kelonia Therapeutics

Gene Drug Developer

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In recent years, the development of cell and gene therapy (CGT) has provided revolutionary treatment methods for people to overcome cancer, rare diseases, autoimmune diseases, etc., but this is stillAn arduous battle requiring multi-party collaboration and united efforts.According to incomplete statistics, 2024Q1 saw 28 collaborations in the CGT field., with the highest total potential valueThe $1.3 billion deal between Novartis and Voyager is based on a new typeCollaboration in gene therapy.

1、AllogeneWith Arbor

On March 12, Allogene Therapeutics and Arbor Biotechnologies jointly announced a non-exclusive global gene editing licensing agreement to apply Arbor's proprietary CRISPR gene editing technology to Allogene's next-generation AlloCAR T™ platform for the treatment of autoimmune diseases.

Allogene designs the next generation of allogeneic CAR-T research products based on its deep understanding of CAR-T research and development. Allogene's first AlloCAR T product for autoimmune diseases is expected to enter Phase I clinical trials in early 2025.Arbor discovers and develops next-generation gene medicines based on its machine learning/artificial intelligence-driven discovery platform for genomic editors.As Arbor continues to advance clinically, with an initial focus on liver and central nervous system diseases, the company is also collaborating with Vertex Pharmaceuticals on several gene-editing and ex vivo cell therapy projects to expand the reach of its novel nuclease technology.

2. Xyphos Biosciences and Kelonia Therapeutics

On February 15, Astellas announced that its wholly-owned subsidiary Xyphos Biosciences and Kelonia Therapeutics had reached a research collaboration and licensing agreement to jointly develop novel cancer immunotherapies.

Under the terms of the agreement, the two companies will combine Kelonia's iGPS® platform with Xyphos' ACCEL TM technology to develop two innovative in vivo CAR-T cell therapies. Xyphos will be responsible for the development and commercialization of the collaborative research products. Kelonia will receive a $40 million upfront payment from the first project. If Xyphos exercises the option for the second project, Kelonia will also receive an additional $35 million, along with potential milestone and contingent payments., with a total amount close to 800 million US dollarsIn addition, Kelonia will receive research and development funding for the work carried out in the collaboration and is eligible to receive tiered royalties in the double-digit percentage range on net sales.

3. Kelonia Therapeutics and Biotron Biotech

On February 19, Kelonia Therapeutics (Suzhou) Co., Ltd. ("Kelonia Therapeutics") and Beijing Better Biotechnology Co., Ltd. ("Better Biotechnology") announced the official signing of a strategic cooperation agreement. In the new year, the two parties will engage in in-depth cooperation in the in vivo CAR-T field, jointly promoting technological innovation and development with an enterprising spirit of exploration, contributing to advancements in the healthcare sector.

Co-founder and General Manager of Kelonia TherapeuticsRen KeyunPh.D.Kelonia Therapeutics has been deeply engaged in the fields of nucleic acid drugs and cell gene therapy, demonstrating a high level of foresight and sensitivity towards innovative solutions. Significant progress has been made in the clinical treatment of in vivo CAR-T, providing a novel solution for unmet therapeutic needs. We believe that with further research and continuous technological advancements, mRNA-LNP technology will create opportunities for CAR-T therapy to be more widely applied in the field of cancer treatment.

4.Immunocore and BMS

On February 22, Immunocore announced a clinical trial collaboration and supply agreement with Bristol Myers Squibb. The two parties will jointly study the efficacy and safety of Immunocore's off-the-shelf T cell receptor (TCR) bispecific therapy IMC-F106C in combination with the PD-1 inhibitor Opdivo (nivolumab) for the first-line treatment of advanced melanoma.

Under the cooperation agreement, Immunocore will be responsible for the funding required for the Phase 3 clinical trial PRISM-MEL-301 of IMC-F106C in combination with Opdivo as a first-line treatment for advanced melanoma, and Bristol-Myers Squibb will be responsible for providing Opdivo.

The PRISM-MEL-301 trial will randomly assign HLA-A*02:01-positive advanced melanoma patients to receive IMC-F106C + Opdivo, Opdivo monotherapy, or Opdivo + relatlimab (LAG-3 Targeting Monoclonal Antibody) combination therapy, depending on the country and region where the patient is enrolled. Immunocore plans to administer the first patient dose in the first quarter of 2024.

5. Kelonia Therapeutics and Crystal Bio

On February 21, CATUG (Kaituo Biotech) and Crystal Bio (a US-based large molecule analytical CRO under Crystal Pharmatech) officially entered into a strategic partnership. They have established the CATUG-Crystal Joint Laboratory in North America to jointly provide high-quality nucleic acid drug analysis services.

Kelonia Therapeutics focuses on providing full-process CRDMO services for nucleic acid drugs and possesses a one-stop technology platform. In collaboration with Crystal Bio's professional large-molecule analytical CRO services, it addresses various complex analytical challenges in CMC, preclinical, and clinical samples in a rapid, efficient, and flexible manner. Together, they are building an internationally competitive nucleic acid drug analysis and testing platform in North America, offering high-quality analytical services to local customers.

CATUG-Crystal Joint Laboratory is located in New Jersey and the Boston area of Massachusetts, aiming to address analytical challenges in the field of mRNA and nucleic acid drug therapies. It provides tailored characterization and comprehensive analytical services for all stages of nucleic acid drug development, enhancing customer experience through professional, rapid-response, and flexible localized services.

6. Intellia Therapeutics and ReCode Therapeutics

On February 16, Intellia Therapeutics and ReCode Therapeutics announced a strategic collaboration to jointly develop novel genomic medicines for the treatment of cystic fibrosis (CF).This collaboration combines Intellia's CRISPR-based platform (including its DNA writing technology) with ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, expanding the scope of gene editing to include pathogenic targets in the lungs.

As part of the agreement, the two companies will focus their initial research efforts on therapy development to address CF issues in patients with limited or no available treatment options, with the opportunity to expand the scope of collaboration at a later stage. Intellia will be responsible for designing the editing strategy and component development for the investigational therapies. ReCode will lead the subsequent preclinical and clinical development. ReCode will also lead the global commercialization of certain projects within the collaboration. Intellia will be eligible to receive predetermined development and commercial milestone payments.

7.BioNTech and Autolus Therapeutics

On February 8, BioNTech and Autolus Therapeutics announced a strategic collaboration aimed at advancing the development and commercialization of their autologous CAR-T cell therapies. Under the agreement, Autolus will receive a $50 million upfront payment, and BioNTech will make a $200 million equity investment in Autolus.

BioNTech to Gain Access to Autolus' Manufacturing Capacity, Clinical Trial Sites, and Commercialization Network in the UK, Accelerating the Development of BioNTech’s Investigational Therapy BNT211 in More CLDN6-Positive Tumor Types. BioNTech Anticipates Including Over 10 Potential Registrational Clinical Trials in Its Pipeline by the End of 2024.AUtolus will lead the development and commercialization of its investigational therapies AUTO1/22 and AUTO6NG in any tumor indications. BioNTech has the option to support certain development activities and co-commercialize these two candidate products in specific regions.

8. Novartis and Voyager

On January 2, Voyager Therapeutics announced that it had signed a capsid licensing agreement with Novartis subsidiary Novartis Pharmaceuticals and established a strategic partnership to advance novel gene therapies for Huntington's disease (HD) and spinal muscular atrophy (SMA).

According to the agreement, Novartis will pay Voyager $100 million, including $20 million for the purchase of newly issued shares of Voyager. Voyager is eligible to receive up to $1.2 billion in preclinical, development, regulatory, and sales milestones, as well as tiered royalties on global net sales of products containing Voyager's TRACER capsid, totaling $1.3 billion.

Novartis will obtain the exclusive rights to the TRACER capsid target related to SMA from Voyager and will be responsible for all development and commercialization. Novartis will also acquire global rights to Voyager's AAV gene therapy for HD, utilizing Voyager’s TRACER capsid and proprietary payload. Voyager will be responsible for preclinical progress, while Novartis will handle all clinical development and commercialization for the HD program.

9. JW Therapeutics and 2seventy bio

January 2, WuXiJunoAnnounced an exclusive collaboration agreement with 2seventy bio to co-develop and commercialize treatments for autoimmune diseases in Greater China.SexChimeric Antigen Receptor (CAR) T-cell therapy products for the treatment of diseases. The strategic alliance is an expansion of the partnership established in 2022 for the MAGE-A4 project, building on the company’s capabilities in cell therapy development, with the initial aim of accelerating progress in Greater China.Development and validation of T-cell-based immunotherapy products. In addition, the contracting parties plan to add other candidate products in the future.

The company will conduct process development and the first-in-human clinical trials in China, with costs shared by both parties. During the specified period, the company has exclusive rights to negotiate an exclusive license for the development, production, and commercialization of products in Greater China. According to the cooperation agreement, the company is also eligible to receive from 2seventy bio development, regulatory, and sales milestones in the high double-digit millions of US dollars, as well as royalties on global net sales (excluding Greater China).

10. Reindeer Bio and Umoja Biopharma

January 4, 2024, Icarus Bio and UmOja Biopharma jointly announced a series of new collaborations on the research, development, and commercialization of novel in vitro and in vivo cell and gene therapies. Previously, in November 2022, Ixotech Biology and Umoja reached an agreement to combine Ixotech Biology’s chimeric antigen receptors (CARs) with Umoja’s iCIL platform to co-develop next-generation off-the-shelf cell therapies. The signing of this new agreement deepens the strategic cooperation between the two parties and further advances innovative off-the-shelf cell and gene therapy solutions.

According to the agreement, Ixobio will obtain the exclusive rights to Umoja's Rapamycin-Activated Cytokine Receptor (RACR™) platform—synthetic cytokine receptor technology—for the development of two in vitro iPSC-derived Chimeric Antigen Receptor (CAR) cell therapies. Ixobio will be responsible for the global research, production, registration, and commercialization of these two in vitro cell therapy products. Meanwhile, Umoja will exclusively use two clinically validated CAR sequences targeting different antigens from Ixobio, combined with Umoja’s self-developed lentiviral vector-based in vivo gene delivery technology—VivoVec™—to advance the development of two in vivo CAR-T candidates. Ixobio and Umoja will separately handle product R&D, manufacturing, registration, and commercialization in Greater China and regions outside Greater China, respectively. Additionally, both parties are entitled to receive corresponding development and sales milestone payments as well as royalties within the collaboration. This partnership will promote the advancement of innovative off-the-shelf cell and gene therapies and apply them in the fields of oncology and autoimmune diseases.

Summary

In the current pharmaceutical environment, actively seeking partners is a common topic for biotech companies, achieving a 1+1>2 outcome through win-win cooperation. As the number of collaboration deals increases, "return" cases are also common, such as this year.In February, BeiGene announced the termination with Shoreline Biosciences.The latter has developed allogeneic NK cell therapies targeting 4 different sites.CombineWork;Everest Medicines Announces Termination of mRNA Therapy Collaboration and License Agreement with Providence Therapeutics, "Return" Mostly Due to Strategic Decisions. This Also Shows That Only by Assessing the Situation,Only by doing the right thing with the right partner at the right time can we achieve a win-win situation.

Reference: Official websites of various companies