Shark Tooth Biotech

What Shark Tooth Biotech does

Shark Tooth Biotech is a San Francisco-based, founder-owned biotechnology company established in September 2024 by David Apple to develop treatments for Charcot-Marie-Tooth Disease Type 1A (CMT1A), a progressive genetic neuropathy for which no treatment or cure currently exists. The company was catalyzed by the founder's personal experience following his son Ari's diagnosis in January 2022 and operates with a stated US-only total addressable market of approximately $14 billion per year under Orphan Disease Designation incentives, serving an addressable patient population of roughly 1.6 million people worldwide.

The company's core offering is the CMT1A Treatment Development Program, built around a "Drug Assembly" methodology rather than de novo drug discovery. Shark Tooth Biotech partners with research laboratories — including ENCell and the National Research Council Canada — to fund studies testing whether existing proprietary RNA-based platforms (antisense oligonucleotides and siRNA chemistries, paired with advanced delivery technologies for the peripheral nervous system) developed for other indications can be repurposed for CMT1A. AI tools are used to accelerate therapeutic candidate design and optimization, and the company's stated longer-term platform thesis extends to roughly 5 million people affected by other peripheral nerve diseases once the CMT1A program is de-risked.

Shark Tooth Biotech is pre-revenue and funds operations through a hybrid capital model that combines equity investments and tax-deductible donations, captured via online inquiry forms and a Blackbaud-powered donation portal. The team is intentionally lean: founder/CEO David Apple leads full-time, supported by a fractional CTO (Wil Chow, with prior experience overseeing 7 IND molecules and 2 NDA drugs across 10 biotechs) and a fractional CSO (Jordan VerPlank, an academic CMT1A researcher). Scientific advisors from Harvard, Johns Hopkins, and leading biotech executives supplement the core team. Strategic community ties include a board seat on the Charcot-Marie-Tooth Association held by the founder and ecosystem support from South Park Commons, with the company distributing updates through LinkedIn, X/Twitter, and a quarterly email newsletter.

Name

Shark Tooth Biotech

Legal name

Shark Tooth Biotech

Website

https://sharktooth.bio

Company type

Private

Founded year

2024

Operating status

Operating

Headcount range

1–10 employees

Short description

Shark Tooth Biotech is a San Francisco-based, founder-owned biotech developing RNA-based treatments for Charcot-Marie-Tooth Disease Type 1A (CMT1A) using a "Drug Assembly" approach that repurposes existing therapeutic technologies from partner labs.

Ownership category

akta.pro rank

Product category

Biotech R&D Services

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Services-Commercial Physical & Biological Research (8731), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)

akta.pro secondary industries

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)

Keywords

• RNA therapeutics
• Rare disease treatments
• Antisense oligonucleotides
• Peripheral neuropathy research
• Drug repurposing

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Markets served

GTM type

B2C

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Investment Capital: The company seeks equity investments from investors to fund R&D operations and clinical development activities for CMT1A treatments.
2. Tax-Deductible Donations: Accepts charitable donations which are tax-deductible, appealing to mission-driven donors supporting rare disease research.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels3 records

Core offering

Shark Tooth Biotech is a pre-revenue biotechnology company developing RNA-based therapeutics targeting Charcot-Marie-Tooth Disease Type 1A (CMT1A), a progressive genetic neuropathy affecting 1.6 million people worldwide. The company's core offering is its CMT1A Treatment Development Program, which employs a "Drug Assembly" approach: rather than building drugs from scratch, Shark Tooth partners with research laboratories (including ENCell and NRC Canada) to fund studies that test whether existing proprietary RNA, ASO, and siRNA technologies proven in other diseases can be applied to CMT1A. The platform leverages AI-accelerated therapeutic candidate design to compress development timelines.

Product overview

Shark Tooth Biotech is a mission-driven biotech company developing treatments for CMT1A (Charcot-Marie-Tooth Disease Type 1A). The company operates a single therapeutic development program using a 'Drug Assembly' approach rather than new drug discovery. This involves partnering with leading labs and funding research to test whether existing technologies (such as RNA therapies, ASO, and siRNA chemistries) that have proven effective in other diseases can be applied to CMT1A. The company leverages advanced AI tools for therapeutic candidate design, evaluation, and optimization. No commercial products have been launched yet as the company is in the research and development stage.

Differentiator

Problem solved

Functional benefit

Products and services

• CMT1A Treatment Development Program A pre-clinical therapeutic development program focused on creating the first treatment for Charcot-Marie-Tooth Disease Type 1A (CMT1A). The program employs a Drug Assembly approach, partnering with research laboratories (ENCell, NRC Canada) to fund and test whether existing proprietary RNA, ASO, and siRNA technologies proven in other diseases can be applied to CMT1A, using AI-accelerated therapeutic candidate design to compress timelines. The intended end-benefit is halting or reversing CMT1A disease progression for the 1.6 million patients worldwide, with subsequent expansion potential to 5 million people affected by peripheral nerve diseases.

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Four partnerships are on record, tiered core and supporting.

• ENCellcoreStrategic or Co-development PartnerPartner laboratory featured on Shark Tooth Biotech's website. ENCell appears to be a research laboratory with proprietary technologies being evaluated for potential application to CMT1A treatment through the Drug Assembly approach.
• NRC CanadacoreStrategic or Co-development PartnerNational Research Council Canada partner featured on Shark Tooth Biotech's website. NRC Canada is a federal agency that conducts research and develops technologies with potential therapeutic applications.
• South Park CommonssupportingStrategic or Co-development PartnerSouth Park Commons is a community of builders and technical founders that provides resources and connections for technology ventures. Partnership appears to provide ecosystem support and potential deal flow.
• CMTAcoreStrategic or Co-development PartnerCharcot-Marie-Tooth Association is a patient advocacy organization. Founder David Apple serves as a Board Member. CMTA provides research funding, patient support, and scientific advisory expertise including collaboration on the CMT1A Ionis paper.

Scale indicators3 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Sarepta Therapeutics: Sarepta is the leading RNA-based rare-disease therapeutics company (PMO/ASO chemistries for Duchenne); a broader incumbent using similar oligonucleotide chemistries that could plausibly extend into CMT1A.
• Alnylam Pharmaceuticals: Alnylam is the established siRNA platform leader whose chemistry Shark Tooth explicitly cites as a model; a broader incumbent but directly comparable on the siRNA modality for neurological indications.
• Biogen: Biogen has deep neuropathy R&D and rare-neurology commercial infrastructure that could be deployed into CMT1A — a broad incumbent that represents both a potential acquirer and a competitive threat.
• BridgeBio Pharma: BridgeBio's subsidiary has an active CMT1A gene-therapy program; as a large, well-capitalized rare-disease biotech, it represents a credible incumbent threat to Shark Tooth's first-mover positioning.

Direct peers

• Ionis Pharmaceuticals: Ionis published the seminal 2017 ASO proof-of-concept reversing CMT1A in mice and remains the most directly comparable ASO-based competitor pursuing CMT1A therapeutics — the same modality Shark Tooth is built around.
• Helixmith: Helixmith is advancing Engensis, a gene therapy for CMT1A/diabetic neuropathy, directly competing for the same CMT1A patient population with a different but overlapping therapeutic modality.
• Pharnext: Pharnext is developing PXT-3003, the most clinically advanced CMT1A therapeutic candidate (in late-stage trials), making it the closest direct competitor by indication even though its approach is small-molecule combination therapy.

Emerging players

• DTx Pharma (Novartis): DTx Pharma (acquired by Novartis) developed siRNA delivery technology specifically for peripheral nervous system targets, making it a directly comparable technical platform addressing the same PNS-delivery challenge Shark Tooth identifies as its core risk.
• InFlectis BioScience: InFlectis is a small, CMT1A-focused biotech developing IFB-088, a small molecule acting on PMP22 — a directly comparable single-indication rare-neuropathy biotech at a similar stage.
• Augustine Therapeutics: Augustine Therapeutics is an emerging neuromuscular-focused biotech; a comparable small, indication-specific rare-neuro disease peer at a similar developmental stage.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/SharkToothBio
• LinkedInlinkedin.com/company/shark-tooth-biotech

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Shark Tooth Biotech