Volume 17 Issue 4, December 2023

NurExone has created ExoTherapy, a cutting-edge exosome-based drug-delivery platform, and is currently developing its lead product, ExoPTEN, as a novel therapy for acute spinal cord injuries.

By engineering naturally existing arrestin domain-containing protein 1 (ARRDC1)-mediated microvesicles (known as ARMMs), Vesigen Therapeutics’ platform enables the effective delivery of novel biotherapeutics to specific tissues and cell types.

As Pandorum Technologies’ bioengineered ‘liquid cornea’, Kuragenx, is about to enter a first-in-human study for vision restoration in patients suffering from corneal blindness, the company is extending its proprietary technology platform to develop advanced exosome-based therapeutics for lung and liver regeneration.

bit.bio is a synthetic biology company that uses its cell identity coding platform to reprogram induced pluripotent stem cells to create any human cell type.

Pennsylvania-based Ocugen is a fully integrated, patient-centric biotech company focused on the development of vaccines in support of public health, and gene and cell therapies targeting unmet medical needs, all based on its three scientific platforms.

UK-based Broken String Biosciences is developing a commercial analytics platform for CRISPR therapeutics safety, a space in growing need of a gold standard solution for measuring off-target gene editing.

With its unique and proprietary genetically modified adipocyte (GMAC) platform, CellGenTech is developing novel ex vivo gene therapies and has achieved the first-in-human study of an adipocyte-based gene therapy.

The biotech firm’s polyclonal anti-cancer therapy is based on an ex vivo approach and avoids the use of toxic immune-depleting chemotherapy.

Companies that have pioneered platforms based on oligonucleotides targeting RNA and their biopharma partners continue to move beyond rare diseases into broader patient populations with cardiovascular diseases and central nervous system disorders.

Philadelphia’s Aro Biotherapeutics has developed a novel RNA platform that leverages Centyrin proteins to deliver oligonucleotides to muscle and immune cells.

Cancer Research Horizons (CRH) is harnessing the skills, expertise, and research network of its parent organization to speed-up the discovery and development of transformative oncology drugs, improve patient outcomes, and advance assets for biopharma partners.

Unique proteolipid vehicle (PLV) technology developed by Entos Pharmaceuticals is broadening the scope for biopharma to deliver nucleic-acid payoffs to target cells throughout the body, paving the way for a new generation of curative drugs.

Primrose Bio’s advanced technology for manufacturing nucleic acids and proteins makes it the partner of choice for pharma companies developing new vaccines, therapeutics and bio-based products.

Through its innovative asymmetric siRNA platforms, OliX Pharmaceuticals is opening up huge opportunities in RNA interference-therapeutic development to address unmet needs for various indications spanning the liver, eye, skin, and beyond.

Azor Biotek is applying artificial intelligence solutions to the design and development of tailored, highly specific, novel small molecules, tapping into the vast potential of RNA drug targets.

Visionary investment in mRNA technology platforms is allowing ST PHARM and its two startups, Levatio Therapeutics and Vernagen, to foster partnerships and advance the development of cutting-edge RNA-based drugs and vaccines.

Genialis is the RNA biomarker company; it espouses a people-first approach to machine learning–driven biomarker discovery. For targeted therapies to benefit the most patients possible, Genialis develops predictive biomarkers to guide treatment decisions. The company’s next-generation biomarkers work for real patients by informing targeted treatment decisions for better outcomes.

Approaches to slow or reverse aging have gained momentum in recent years, but the field is still in its infancy with hurdles to overcome, as illustrated by efforts to develop therapies that clear senescent cells.

Rejuveron’s breakthrough therapeutics are set to transform the treatment of age-related diseases by targeting the root causes of aging.

With its first-in-class Cholesterol Degrading Platform (CDP), Repair Biotechnologiesis tackling the root cause of many difficult-to-treat age-related diseases.

BioViva Science is a gene-therapy company focused on treating aging-related complex diseases with a new gene-therapy platform.

NIBEC (the Nano Intelligent Biomedical Engineering Corporation)’s transformative carrier platform is delivering macromolecular drugs to cell and tissue targets throughout the body with improved therapeutic efficacy and reduced side effects.

Deciduous Therapeutics is developing a novel class of immuno-therapies designed to systemically clear senescent cells and to treat co-morbidities of aging.

An evolving regulatory environment is encouraging biopharma companies to invest more heavily in therapies for central nervous system diseases.

Acumen Pharmaceuticals is developing ACU193 as the first clinical-stage therapeutic to selectively target toxic soluble amyloid-β oligomers (AβOs), which are an early and persistent driver of Alzheimer’s disease-related neurodegeneration.

By taking a neuroanatomy-led gene-therapy approach, AviadoBio is developing targeted drugs and delivery technologies to advance the treatment of neurological conditions such as frontotemporal dementia and amyotrophic lateral sclerosis.

Based on advances in the understanding of the etiology of neurodegeneration, SciNeuro Pharmaceuticals is developing ground-breaking therapies with multi-indication potential.

We review the highest-value deals of the year with the help of DealForma.

By combining molecular science and artificial intelligence with years of experience and a world-leading database, Caris Life Sciences aims to revolutionize cancer care and deliver on the promise of precision oncology medicine in partnership with biopharma and industry innovators.

Achelois BioPharma has built a screening platform to discover T cell-intrinsic genetic-enhancing modifiers that enable tumor-specific T cells to eliminate established tumors in preclinical models and patients.