Cabaletta Bio, Inc. is a clinical-stage biotechnology company developing engineered T cell therapies targeting autoimmune diseases involving B cells.

# Company Summary: Cabaletta Bio, Inc.

## Business Focus
- Cabaletta Bio, Inc. is a clinical-stage biotechnology company developing engineered T cell therapies targeting autoimmune diseases involving B cells.
- The company's proprietary platform, called **CABA® (Cabaletta Approach to B cell Ablation)**, includes two main strategies:
  - **CARTA (Chimeric Antigen Receptor T cells for Autoimmunity):** Designed to reset the immune system.
  - **CAART (Chimeric Autoantibody Receptor T cells):** Engineered to selectively eliminate disease-causing B cells.

## Current Product Candidate
- **Rese-cel (resectabtagene autoleucel):**
  - A fully human CD19-CAR T construct with 4-1BB co-stimulatory domain.
  - Designed to achieve transient, deep depletion of B cells with a single infusion.
  - Aims to reset the immune system, providing durable responses.
  - Intended for autoimmune diseases such as Systemic Lupus Erythematosus (SLE), Lupus Nephritis, Myositis, Systemic Sclerosis (SSc), Myasthenia Gravis (MG), Multiple Sclerosis (MS), Pemphigus Vulgaris (PV), among others.
- **Development Milestones:**
  - FDA granted clearance for IND applications for rese-cel in multiple indications including SLE, myositis, SSc, gMG, and MS.
  - Received Fast Track Designation from the FDA for rese-cel in several indications.
  - As of March 14, 2025, 33 patients enrolled in clinical trials across 56 active clinical sites in the U.S. and Europe.

## Clinical Trials
- Conducts several Phase 1/2 clinical trials (RESET™ trials) in indications such as:
  - SLE & Lupus Nephritis
  - Myositis
  - Systemic sclerosis
  - Generalized Myasthenia Gravis
  - Pemphigus Vulgaris
  - Multiple Sclerosis
- These trials are designed as independent cohorts with 6 patients per cohort, aiming for broad investigation across diseases.
- Positive early data with reductions in disease markers and no serious adverse events observed in initial patients.

## Manufacturing Strategy
- Uses partnerships with academic centers (Penn, CHOP) and multiple Contract Development and Manufacturing Organizations (CDMOs) (WuXi, Oxford, Lonza) for process development and supply.
- Collaborated with Cellares for automated manufacturing technology.
- Plans to build internal manufacturing capacity, but currently relies on external partners.

## Employees & Operations
- As of December 31, 2024:
  - **Total Employees:** 164
  - **Research & Development Employees:** 135
  - **Locations:** Headquarters in Philadelphia, PA (2929 Arch Street, Suite 600); subsidiaries in Switzerland and Germany.
  - No employees are part of a union.
- No revenue or net income figures provided in the document. The company is in the development stage and has incurred significant losses to date.

## Market & Financials
- As of June 30, 2024, the company's estimated market value (market capitalization) was approximately **$350 million** based on publicly reported stock price.
- Shares outstanding as of March 27, 2025: **50,743,101**.

## Summary Notes
- The company is focused on developing engineered T cell therapies for autoimmune diseases.
- Product candidates are in clinical trials, with no commercial products yet.
- The company has a strategic manufacturing partnership and relies heavily on external manufacturing with plans for internal capacity.
- No specific revenue, profit, or customer numbers are provided—business is in early development with ongoing clinical trials.

Cabaletta Bio, Inc. is a clinical-stage biotechnology company pioneering engineered T cell therapies for autoimmune diseases.

# Company Overview: Cabaletta Bio, Inc.

## Business Focus
Cabaletta Bio, Inc. is a clinical-stage biotechnology company dedicated to the discovery and development of innovative engineered T cell therapies. The company aims to provide deep and durable, potentially curative responses to patients suffering from autoimmune diseases with a one-time administration of their therapies.

## Proprietary Platforms
Cabaletta operates through its proprietary platform called CABA™ (Cabaletta Approach to B cell Ablation), which features two main strategies:
1. **CARTA (Chimeric Antigen Receptor T cells for Autoimmunity)**: Aims for transient and complete depletion of all B cells to potentially reset the immune system.
2. **CAART (Chimeric AutoAntibody Receptor T cells)**: Tailored to selectively eliminate disease-causing B cells while sparing healthy ones.

## Product Candidates
- **CABA-201**: The lead product candidate targeting a broad range of autoimmune diseases, designed to eliminate CD19-positive B cells.
- **DSG3-CAART**: A CAAR T cell therapy targeting mucosal pemphigus vulgaris (mPV).
- **MuSK-CAART**: Aiming at treating muscle-specific kinase (MuSK) myasthenia gravis (MG).

## Clinical Trials
Cabaletta is actively conducting multiple clinical trials:
- **RESET-SLE™ Phase 1/2** (for systemic lupus erythematosus): Actively enrolling patients.
- **RESET-Myositis™ Phase 1/2** (for myositis): Actively enrolling patients.
- **RESET-SSc™ Phase 1/2** (for systemic sclerosis): IND application cleared, trial launching soon.
- **RESET-MG™ Phase 1/2** (for generalized myasthenia gravis): IND application cleared, trial launching soon.

## Financial Overview
As of December 31, 2023:
- **Employees**: 103 full-time employees
- **Revenue**: The company has not generated revenue as it is still in the clinical development stage.
- **Net Loss**: $67.7 million for the year ended December 31, 2023.
- **Accumulated Deficit**: $233.2 million.
  
## Market Potential
- **Target Market**: The company focuses primarily on patients with autoimmune diseases, with significant unmet medical needs.
- **Estimated Patients**: SLE alone affects an estimated 160,000-320,000 patients in the U.S.

## Research Collaboration
Cabaletta has partnerships with leading academic institutions, particularly the University of Pennsylvania, to support its research and development activities, leveraging their expertise in CAR T technologies and autoimmunity.

## Competitive Landscape
The company operates in a competitive landscape that includes several large pharmaceutical and biotechnology companies working on CAR T and other engineered T cell therapies. However, clinical-grade engineered T cell therapies for autoimmune diseases remain largely undeveloped, presenting Cabaletta with a first-mover advantage in this niche. 

## Risk Factors
Despite its promising approach, Cabaletta faces various risks, including clinical trial failures, regulatory delays, challenges in manufacturing, and the potential for adverse side effects from its therapies. The company's financial viability depends largely on successful clinical outcomes and eventual commercialization of its therapies. 

## Summary
Cabaletta Bio, Inc. is pioneering engineered T cell therapies for autoimmune diseases through its CABA™ platform. With a dedicated team and innovative approach, the company aims to address significant unmet medical needs while navigating a complex regulatory environment and challenging market landscape.

Cabaletta Bio, Inc.

10-K / March 31, 2025

10-K / March 21, 2024

10-K / March 31, 2025

Company Summary: Cabaletta Bio, Inc.

Business Focus

Current Product Candidate

Clinical Trials

Manufacturing Strategy

Employees & Operations

Market & Financials

Summary Notes