Top 10 biotech startups

10: TreeFrog Therapeutics

Backed with funding from the European Union’s Horizon 2020 Research and Innovation Program, TreeFrog Therapeutics is a stem cell startup company. 

In its efforts to provide millions of patients with access to cell therapies, TreeFrog Therapeutics has developed C-Stem, an end-to-end 3D scalable solution to dramatically reduce treatment costs. Alongside its strategic partners, TreeFrog Therapeutics’ ambitions are to secure production and quality, fasten clinical development and facilitate access in the market. 

09: SparingVision

Focused on the discovery and development of genomic medicines to address serious ocular disorders, SparingVision’s lead product is its breakthrough therapy for retinitis pigmentosa (RP), a retinal disease affecting almost two million people around the world. 

SparingVision’s SPVN06 product uses “a gene therapy-based approach agnostic of mutated genes,” and is delivered via a single subretinal injection to prevent the degeneration of photoreceptors. So far SparingVision has raised €44.5mn in funding.

08: Poseida Therapeutics

Leveraging its gene engineering platform technologies, Poseida Therapeutics is a clinical-stage biopharmaceutical company that strives to bring better treatments to patients with serious diseases. 

Poseida Therapeutics’ four-gene engineering platforms include piggyBac, CAS-Clover, Gene Delivery, and CAR-T Tools to provide a safer, more durable, and more efficient suite of treatments.

Poseida Therapeutics became public in July 2020, and is a spinout organisation from its parent company, Transposagen, an early leader in the development of gene engineering technology.

07: Beam Therapeutics

DNA has four types of bases: adenine (A), cytosine (C), guanine (G), and thymine (T). The human genome consists of more than three billion pairs (adenine and thymine, and cytosine and guanine). If point mutation occurs in the genome, proteins can become dysfunctional or missing altogether which causes diseases.

Beam Therapeutics, a pioneer in the use of CRISPR base editing, is harnessing its groundbreaking technology to “make permanent, specific edits to single bases in DNA and RNA, without cutting the strands.” In doing so, Beam Therapeutics is developing a new class of precision-genetics medicine by “combining precision targeting of the genome with precision control of editing outcomes.” 

06: Nouscom

Led by successful entrepreneurs with prior experience in developing innovative candidate vaccines for infectious diseases, Nouscom’s platform technology is based on recombinant Adenovirus isolated from non-human great apes (GAd) and Modified Vaccinia Virus Ankara (MVA). 

Evaluated in more than 4,000 adults, newborns, and elderly healthy subjects, as well as those with chronic infectious diseases, Nouscom states that the vaccine therapy has shown to be safe and effective, and will continue to leverage its expertise and experience to further develop its therapies.

05: GRAIL

Combining science, technology and population-scale clinical studies, GRAIL’s mission is to detect cancer early in patients. Through continuous collaboration, high intensity sequencing, and modern data science, GRAIL is working to create vast datasets to develop evidence that supports its products.

Such projects include the development of a blood test which combines high-intensity genomic sequencing and complex computational algorithms to find cancer-specific patterns to detect multiple types of cancer.

04: BioSplice (formerly Samumed)

Since its founding in 2008, BioSplice (formerly Samumed) has had a unique operating philosophy based on building a broad technology platform for modulating regenerative pathways to improve patient health.

To address degenerative diseases, one of BioSplice’s primary signaling pathways is the Wnt pathway, which regulates the self-renewal and differentiation of adult stem cells. “The ability to modulate the Wnt pathway, and thereby recover and restore the health of diseased tissues, presents significant opportunities in regenerative therapeutics,” says BioSplice. 

03: Ginkgo Bioworks

Believing that biology is “the best manufacturing technology on the planet,” Ginkgo Bioworks collaborates with its partners and ecosystems, to automate and scale the organism engineering process, providing engineers with the ability to prototype thousands of biological designs.

Ginkgo Bioworks’ five areas of expertise include strain improvement, enzyme discovery, new product development, biosecurity, and mammalian cell engineering. 

“The interesting thing to program in the 21st century isn’t going to be computers - it’s biology,” says Tom Knight, Co-Founder of Ginkgo Bioworks.

02: Passage Bio

Passage Bio, a genetic medicines company that develops transformative therapies for the treatment of rare monogenic central nervous system (CNS) disorders. Putting patients at the centre of every decision made, its vision is to become the premier genetic medicines company that dramatically and positively transforms the lives of patients.

To achieve this, Passage Bio is in collaboration with the University of Pennsylvania’s Gene Therapy Program (GTP), gaining access to cutting edge capabilities and innovation in genetic medicine research.

Passage Bio has three lead product candidates, as well as three ongoing discovery programs, and options to license eleven additional programs from the GTP. Passage Bio purposefully focuses on rare, monogenic CNS as it believes its genetic medicine approach provides “distinct technical advantages based on decades of research by GTP.”

01: Cyclerion Therapeutics

With its team of determined experts to develop innovative medicines, Cyclerion Therapeutics is a clinical-stage biopharmaceutical company, focused on “unlocking the full therapeutic potential of the NO-cGMP pathway,” addressing the broad range of serious central nervous system (CNS) diseases.

At the forefront of CNS drug development, Cyclerion Therapeutics’ systems biology approach combines genetic and proteomic data to identify CNS diseases. Its approach identifies dysfunctional NO-sGC-cGMP signaling as the common pathophysiologic thread for both neurodegenerative diseases and neuropsychiatric diseases.

“In diseases associated with deficits in the NO-sGC-cGMP pathway, CY6463 [a sGC stimulator] has the potential to address the underlying pathophysiology by restoring appropriate endogenous signaling and maintaining the precise spatial and temporal control that is the hallmark of this signaling pathway,” says Cyclerion Therapeutics.