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splicebio

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SpliceBio Market Research Report



Overview



SpliceBio is a pioneering biotechnology firm focused on gene therapies, utilizing a proprietary Protein Splicing platform. Established in Barcelona, Spain, SpliceBio is headquartered there and employs a workforce of 11 to 50 people. This innovative platform was honed from over two decades of research in intein and protein engineering at the Muir Lab, Princeton University. The company's focus primarily targets diseases caused by mutations in large genes, with a significant project being the SB-007 drug for Stargardt Disease, which has gained U.S. FDA IND clearance to proceed into Phase 1/2 clinical study.

Recent Developments



Recently, SpliceBio successfully secured €50 million through an oversubscribed Series A financing round, boosting development of its Protein Splicing platform and gene therapy pipeline, indicating robust investor confidence. Further highlighting their strategic progression is a collaboration with Spark Therapeutics, aimed at creating a gene therapy for inherited retinal diseases, relying on the exclusive rights to SpliceBio's technology.

Leadership Team



  • Miquel Vila-Perelló, Ph.D. - Co-Founder & CEO

With a robust background in biotechnology, chemical biology, and protein engineering, Miquel leads SpliceBio's strategic initiatives.

  • Silvia Frutos, Ph.D. - Co-Founder & CTO

Silvia brings her expertise in chemical biology and protein chemistry to the forefront, managing the technological trajectory of the company.

  • Gerard Caelles - Chief Business Officer

Since 2016, Gerard has spearheaded business development, leveraging his experience in biotech company-building to drive strategic growth.

  • Aniz Girach, MD - Chief Medical Officer

A veteran ophthalmologist with 25+ years of experience, Aniz, who joined SpliceBio in 2024, brings specialized knowledge in medical retina and genetic therapy focused on inherited retinal diseases.

Board of Directors



  • Jean-Philippe Combal - Chairman

  • Erica Whittaker - Board Member

  • Jordi Xiol - Board Member

  • Ed Mathers - Board Member

  • Arthur Franken - Board Member

  • Beat Steffen - Board Member

  • Miquel Vila-Perelló - Board Member


Strategic Initiatives and Collaborations



SpliceBio's dedication to innovation in gene therapy, exemplified by the Protein Splicing platform, is further reinforced by their strategic collaboration with Spark Therapeutics. This partnership focuses on addressing genetic vision disorders, widening the impact and reach of their therapeutic offerings. Such moves are a testimony to their growth potential and industry influence, as demonstrated by their success in securing substantial funding and partnerships with key players.

Upcoming Engagements



SpliceBio plans to be actively involved in key scientific and investor conferences from April to June 2024, underscoring their initiative to engage stakeholders and cement their stature as leaders in genetic medicine.

Contact Information



  • Location: SPLICEBIO, SL Parc Científic de Barcelona, Baldiri Reixac, 10, 08028 Barcelona, Spain


  • Contact Email: info@splice.bio


Competitor Profiling



Key Competitors



1. Applied Genetic Technologies Corporation (AGTC)
  • Headquarters: Alachua, Florida, USA

  • Expertise: Gene therapy treatments, focusing on rare ophthalmologic diseases using a proprietary platform for AAV-based therapies.

  • Strategic Alliances: Partnered with Bionic Sight for advancing optogenetic therapies, with the company acquired by Syncona in December 2022.


2. Stargazer Pharmaceuticals
  • Focus: Novel treatments for rare ocular diseases, emphasizing Stargardt Disease. Recently initiated a Phase 2a trial with Series A funding of $57 million from Novo Holdings and venBio Partners.


3. Generation Bio
  • Location: Cambridge, Massachusetts

  • Specialization: Non-viral gene therapy platform for durable, redosable treatments with scalable DNA manufacturing processes.

  • Strategic Investments: Post-IPO equity round secured $76 million, benefitting from technological partnerships, notably with Moderna for T-cell delivery.


4. AAVantgarde Bio
  • Headquarters: Milan, Italy

  • Concentration: Gene therapies for inherited retinal disorders utilizing proprietary AAV vector platforms, with advancements to Phase 1/2 trials backed by a $65.3 million Series A round, supported by Atlas Venture.


Strategic Opportunities for SpliceBio



The competitive landscape offers various strategic paths for SpliceBio:
  • Collaborative Development: Forge research partnerships similar to AGTC’s, enhancing product development capabilities.

  • R&D Investment: Sustain heavy investments in R&D to remain in the forefront of technological advancements.

  • Clinical Advancements: Emulate competitors like Stargazer and AAVantgarde by intensifying participation in advanced clinical trials.

  • Market Differentiation: Create unique technologies or focus on innovative applications within genetic medicine, paralleling efforts seen in Generation Bio’s non-viral strategies, to stand out in the competitive marketplace.

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