In the ever-evolving world of cell and gene therapy, innovation continues to redefine possibility. After a couple of whirlwind years in the industry, I wanted to revisit this article I introduced you to back when I started at Charlton Morris, discussing five promising start-ups in the field. It showcased the work being done at Neogene Therapeutics, Mnemo Therapeutics, Cabaletta Bio, Orgenesis & Deep Genomics.
As expected, these start-ups have been on incredible journeys over the past few years, forging forward despite uncertain times. As the market settles and we look towards 2024 I revisited these companies to find out where they’re up to, and what’s to come.
Whilst AstraZeneca acquired Neogene Therapeutics for an impressive $200m, Orgenesis partnered with Savicell to license and deploy its ImmunoBiopsy™ Platform. Shortly before this, I caught up with Vered Caplan, Orgenesis’s COO who holds a large role in this exciting transition. We discussed the important role of POC technologies in driving forward innovation in CGT markets. Listen to the full episode below:
Meanwhile, Mnemo Therapeutics announced the publication of two groundbreaking scientific studies developed with Institut Curie, Deep Genomics unveiled BigRNA, an AI foundation model that represents a significant advancement in AI for RNA biology and therapeutics, and Cabaletta Bio received FDA Clearance for an IND Application for the Treatment of Systemic Sclerosis with CABA-201.
Looking forward, I want to highlight five newer companies in the cell and gene therapy space worth keeping an eye on. I hope that in two more years, I’ll be back again to showcase more on their exciting journey.
Leucid Bio, based in London, is making waves in the cell and gene therapy landscape. The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has recently granted them clinical trial authorisation (CTA) for their groundbreaking lateral CAR T-cell therapy, LEU011. What sets Leucid Bio apart is its commitment to pushing the boundaries of cancer therapy research. LEU011, targeting NKG2D ligands found in over 80% of human tumours, has received an innovative medicine designation from MHRA, reflecting its potential to revolutionise the treatment of solid tumours. As they embark on the AERIAL trial, Leucid Bio is undoubtedly one to watch, with the potential to make significant contributions to cell and gene therapy.
Kyverna Therapeutics is at the forefront of revolutionising cell therapy for autoimmune diseases. Their groundbreaking platform, Ingenui-T, is set to redefine patient treatment experiences by lowering costs while delivering effective therapies. Partnering with ElevateBio, Kyverna's commitment to innovation shines through as it harnesses cutting-edge manufacturing processes. KYV-101, an autologous anti-CD19 CAR T-cell construct, shows immense promise for B cell-driven autoimmune diseases. With reduced immunogenicity and cytokine-driven side effects, Kyverna Therapeutics is poised to transform how autoimmune diseases are treated. Its dedication to innovation and strategic partnerships puts it on the radar, as it paves the way for a future with improved patient care and transformative therapies.
XNK Therapeutics is forging forward in the field of cancer immunotherapy. Recently granted funding by Sweden's innovation agency, Vinnova, through the Competence Center for Next-generation NK Cell-based Cancer Immunotherapy (NextGenNK), XNK is on the cutting edge of autologous NK cell-based cell therapy development. XNK’s commitment to advancing cancer treatment is evident through its pipeline, which spans haematological malignancies and solid tumour indications, including its most advanced product, evencaleucel, currently in phase II studies. With world-renowned NK cell experts and an in-house GMP facility, XNK Therapeutics is leading the way in developing new and more effective treatments for cancer patients. In a dynamic and evolving landscape, XNK Therapeutics' dedication to innovation and collaboration makes them a company poised to make significant contributions to the field of cell and gene therapy.
Committed to pioneering treatment, Kriya Therapeutics works in collaboration with Everads Therapy, to harness cutting-edge suprachoroidal delivery technology for retinal diseases commitment to pioneering treatments. Kriya's approach targets the complement cascade to address geographic atrophy, holding immense promise in potentially reducing patient burden and providing long-lasting efficacy. The collaboration aligns with a vision to optimise AAV delivery to the eye through one-time administration, a significant leap forward in patient care. With the support of experts and innovative technology, Kriya Therapeutics stands poised to make a meaningful impact in the treatment of retinal diseases and beyond.
Making waves in the field of gene therapy, Voyager Therapeutics is working in partnership with Sangamo Therapeutics to explore a groundbreaking approach to tackle prion disease, a devastating condition with no current treatment options. Leveraging their epigenetic regulation platform and innovative capsid technology, Sangamo and Voyager aim to specifically target and block the expression of the prion protein, a critical factor in prion disease progression. This collaboration reflects the importance of teamwork in addressing challenging medical conditions. Voyager's dedication to developing therapies for diseases of the central nervous system, alongside its strategic partnerships and cutting-edge technology, further supports its innovative approach to gene therapy. With the potential IND submission for a prion disease treatment in 2025, Voyager Therapeutics is undoubtedly at the forefront of revolutionising gene therapy for neurological disorders and beyond.
I truly believe the future of healthcare lies in the innovative work of companies like these, with new and exciting opportunities for people looking to lead the way in how we treat and manage diseases at the forefront of their growth plans.
Are you interested in exploring any of these, or other similar exciting news in the CGT market in some more depth? Click here to join my community of over 3,500 like-minded leaders for exclusive talent, trend, and market insight straight to your inbox every week. Equally, email me below or connect with me on LinkedIn, here, to chat.
[Original article written in September 2021 and updated in November 2023 by Spencer Knight]
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