What Mahzi Therapeutics does

Mahzi Therapeutics Inc. is a clinical-stage, venture-backed biotechnology company founded in 2020 and headquartered in South San Francisco, California (with corporate domicile in California). The company is exclusively focused on developing precision genetic medicines for under-served rare genetic neurodevelopmental disorders, an area where no approved disease-modifying therapies exist. The company's name derives from the Greek word for "Together" and reflects its operating philosophy of uniting patient advocacy groups, academic researchers, industry partners, and an internal team of rare-disease drug development experts around a single therapeutic mission.

The core technology is an AAV9-based gene replacement therapy platform using intracerebroventricular (ICV) delivery directly to the central nervous system, supplemented by antisense oligonucleotide (ASO) approaches for select indications. The lead asset, MZ-1866, is an AAV9 vector delivering TCF4 isoform B under multimer E box regulatory sequences and is being evaluated in the Phase 1/2 UNITE trial for Pitt Hopkins syndrome (NCT07135050). The second program, an AAV9-based WWOX gene replacement therapy for WOREE/SCAR12 disorders, is licensed from the Aqeilan Lab at Hebrew University of Jerusalem and recently produced the world's first human dose in an 8-month-old WOREE infant. A third pre-clinical program uses an ASO approach for CHD2 deficiency in collaboration with the Ulitsky Lab at the Weizmann Institute of Science.

Mahzi is a pre-revenue company with no approved products; its economics are loss-funded by equity capital. It has raised approximately $4M in a Series A led by HBM Partners (October 2022), ~$52.6M via an exempt securities offering disclosed in an SEC Form D filing (July 2024), and $4M in non-dilutive grant funding from the California Institute for Regenerative Medicine (May 2025). The investor base includes Venrock, HealthCap, Droia Ventures, HBM Healthcare Investments, ArrowMark Partners, Ultragenyx Pharmaceutical, and Mitsui Global Investment. The company employs approximately 14 named team members across executive, clinical operations, regulatory, nonclinical, technical operations, and business operations functions. Future revenue, if any, is expected to derive from out-licensing, partnership economics, or commercialization of approved gene therapies targeting ultra-orphan patient populations of a few thousand to tens of thousands of individuals.

Name

Mahzi Therapeutics

Legal name

Mahzi Therapeutics Inc.

Website

https://mahzi.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

11–50 employees

Short description

Mahzi Therapeutics is a clinical-stage biotechnology company developing AAV9 gene replacement and antisense oligonucleotide therapies for ultra-orphan rare genetic neurodevelopmental disorders, including Pitt Hopkins syndrome (lead asset MZ-1866 in Phase 1/2), WOREE/SCAR12, and CHD2 deficiency.

Ownership category

akta.pro rank

Headquarters

HQ city

San Mateo

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pre-revenue / Clinical-stage biotech: Mahzi is a clinical-stage biotechnology company without commercial product revenue. Capital is raised through exempt securities offerings (e.g., $52.6M Form D filing in 2024) and Series A venture funding (e.g., $4M in October 2022). Future revenue would derive from out-licensing, partnerships, or commercialization of approved gene therapies for rare neurodevelopmental disorders.
2. Venture Capital / Equity Financing: Equity-based funding from biotech-focused investors (HBM Partners, Venrock, HealthCap, Droia Ventures, ArrowMark Partners, Mitsui Global, Ultragenyx) and non-dilutive grants (e.g., California Institute for Regenerative Medicine).

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

Mahzi Therapeutics is a clinical-stage biotechnology company developing a focused pipeline of disease-modifying gene and oligonucleotide therapies for ultra-orphan rare genetic neurodevelopmental disorders. Its lead asset, MZ-1866 (AAV9-TCF4 gene replacement therapy), is in the Phase 1/2 UNITE study for Pitt Hopkins syndrome via intracerebroventricular delivery, supported by pre-clinical AAV9-WWOX and CHD2 ASO programs and a Compassionate Use/Expanded Access pathway.

Product overview

Mahzi Therapeutics is a clinical-stage biotechnology company developing a focused pipeline of precision gene and oligonucleotide therapies for under-served rare genetic neurodevelopmental disorders. The portfolio is organized around a core lead asset, MZ-1866 (an AAV9-TCF4 gene replacement therapy for Pitt Hopkins syndrome currently in the Phase 1/2 UNITE study), complemented by a pre-clinical AAV9-based WWOX gene replacement therapy for WOREE/SCAR12 (licensed from Hebrew University of Jerusalem's Aqeilan lab) and a pre-clinical antisense oligonucleotide (ASO) program for CHD2 deficiency developed with the Weizmann Institute's Ulitsky lab. Together with a Compassionate Use / Expanded Access program, these constitute Mahzi's unified therapeutic offering, developed in collaboration with academic research partners and patient foundations.

Differentiator

Problem solved

Functional benefit

Products and services

• MZ-1866 (AAV9-TCF4 Gene Replacement Therapy) Novel AAV9-based gene replacement therapy constructed by inserting TCF4 isoform B (the longest known isoform and one of the most abundant brain isoforms) into an AAV9 expression cassette under regulation of multimer E box sequences. Delivered as a single intracerebroventricular dose for the treatment of genetically confirmed Pitt Hopkins syndrome (TCF4 deficiency). Currently in Phase 1/2 UNITE clinical trial (NCT07135050).
• WWOX Gene Replacement Therapy (AAV9) AAV9-based gene replacement therapy developed in collaboration with the Aqeilan Lab at Hebrew University of Jerusalem to treat WWOX-related neurodevelopmental disorders, including WOREE (WWOX-related epileptic encephalopathy) and SCAR12 (spinocerebellar ataxia 12), caused by autosomal recessive or compound heterozygous mutations in the WWOX gene. Currently pre-clinical; first-in-human dose administered under compassionate use in 2026.
• CHD2 Deficiency ASO Therapy Antisense oligonucleotide (ASO) approach for the treatment of CHD2 deficiency, developed in collaboration with the Ulitsky Lab at the Weizmann Institute of Science, Rehovot. CHD2 deficiency is caused by mutations in the CHD2 gene encoding Chromodomain DNA Helicase Protein 2, an epigenetic regulator, and presents with refractory epilepsy and global developmental delays in children aged 6 months to 4 years. Pre-clinical stage.
• Compassionate Use / Expanded Access Program Formal program providing access to Mahzi's investigational medicines outside of clinical trials for patients with rare neurogenetic disorders who have no other treatment alternatives. Requests are submitted via licensed physicians to [email protected] and reviewed based on local regulatory requirements, stage of clinical development, supply constraints, internal policies, and medical/scientific assessments.

Quantifiable outcome

• First patient in the world dosed with WWOX gene replacement therapy (WOREE syndrome, May–June 2026); clinically stable one month post-treatment with no recurrence of severe seizures
• +2 more outcomes

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Six partnerships are on record, tiered flagship and core.

• University of California San Diego (Muotri Lab)flagshipStrategic or Co-development Partner · 25 February 2026Co-developed MZ-1866, Mahzi's lead AAV9-TCF4 gene replacement therapy for Pitt Hopkins syndrome. Collaboration with Prof. Alysson Muotri at UCSD produced the TCF4 isoform B / AAV9 expression cassette under multimer E box regulatory sequences that constitutes the company's lead clinical asset.
• Schneider Children's Medical Center of IsraelcoreImplementation/ SI/ Consulting Partner · 1 January 2026Clinical site where the world's first WWOX gene therapy (AAV9-WWOX) was administered to an 8-month-old infant with WOREE syndrome, in Petah Tikva, Israel. Functions as a key clinical investigation site for Mahzi's WWOX program.
• CTI Clinical Trial & Consulting ServicescoreImplementation/ SI/ Consulting Partner · 1 July 2025Collaboration with CTI and the Pitt Hopkins Research Foundation to accelerate clinical development of MZ-1866 for Pitt Hopkins syndrome, providing clinical trial operations and consulting expertise for the UNITE Phase 1/2 study.
• Pitt Hopkins Research Foundation (PHRF)coreGTM or Marketing Partner · 1 July 2025Non-profit foundation dedicated to accelerating research and treatments for Pitt Hopkins syndrome. Co-announced the UNITE clinical trial with Mahzi and CTI; distributes community letters to patient/family networks; President Audrey Davidow quoted on clinical milestones.
• Weizmann Institute of Science (Ulitsky Lab)coreStrategic or Co-development Partner · 1 January 2022Collaboration with Prof. Igor Ulitsky at the Weizmann Institute, Rehovot, to develop an antisense oligonucleotide (ASO) approach for the treatment of CHD2 deficiency. Earlier-stage pre-clinical program.
• Hebrew University of Jerusalem (Aqeilan Lab)flagshipStrategic or Co-development Partner · 1 January 2021Co-developed Mahzi's AAV9-based WWOX gene replacement program for WOREE/SCAR12 disorders. Led by Prof. Rami Aqeilan after over a decade of WWOX research. The therapy (including the world's first ICV WWOX gene therapy administered to a human infant) is licensed from Hebrew University to Mahzi.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• Ultragenyx Pharmaceutical: Public commercial-stage rare-disease gene therapy company; a disclosed Mahzi investor and the most direct strategic comparable given overlapping focus on rare genetic disorders and AAV-based modalities.
• uniQure: Established clinical/commercial-stage AAV gene therapy company with CNS programs; a relevant comparable for AAV manufacturing scale-up, regulatory pathway, and commercial-launch economics.
• Regenxbio: Clinical/commercial-stage AAV gene therapy platform company; comparable as a licensor/developer of AAV delivery technology used by rare-disease CNS programs.
• BioMarin Pharmaceutical: Established rare-disease biopharma with multiple approved enzyme replacement and gene therapy products; relevant comparable for orphan pricing, payer negotiation, and global commercial-launch models.

Direct peers

• Taysha Gene Therapies: Clinical-stage gene therapy company developing AAV-based therapies for rare neurodevelopmental and CNS disorders, directly comparable to Mahzi's AAV9-ICV pipeline strategy and ultra-orphan focus.
• Passage Bio: Clinical-stage AAV-delivered gene therapy company targeting rare monogenic CNS disorders; comparable delivery modality and rare neurodevelopmental indication focus.
• Denali Therapeutics: Clinical-stage biotech developing therapeutics that cross the blood-brain barrier for rare neurological diseases; a peer in CNS-targeted rare-disease drug development, though Denali uses engineered protein and small-molecule approaches rather than AAV.
• Stoke Therapeutics: Clinical-stage biotech developing antisense oligonucleotide (ASO) therapies for severe genetic diseases affecting the CNS; directly comparable to Mahzi's CHD2 ASO program and ICV approach to neurodevelopmental disorders.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for severe neurological diseases; directly comparable in AAV-CNS modality, ultra-orphan focus, and rare neuro indication pipeline strategy.

Emerging players

• Prevail Therapeutics (Eli Lilly): Gene therapy company (acquired by Eli Lilly) focused on neurodegenerative diseases including CNS-targeted AAV programs; relevant comparable for AAV gene therapy targeting rare neurological indications.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/mahzi

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Funding overview

Funding rounds3 records

Investors5 records

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M&A

Investments

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

Frequently asked questions about Mahzi Therapeutics