LogicBio Therapeutics Research Ltd.

www.logicbio.com

High-Tech Company

Acquired by Alexion

TECH VERTICALS

Biologicals, Biotechnology, Gene Therapy, Life Sciences, MedTech, Therapeutics

General Data

Sector	Biomed
Company Stage:	R&D
Established	2014
Number of Employees	30
Reg. Number:	abCDe

DESCRIPTION

LogicBio develops lifelong cures for serious, early-onset rare diseases by combining the best of gene therapy and gene editing in a one-time treatment.
A preclinical-stage gene-therapy company with a mission to develop cures for life-threatening diseases, founded by pioneers in gene therapy from leading academic institutions including Stanford University, Tel Aviv University, and Children’s Medical Research Institute in Australia – The company's core platform includes synthetic gene-therapy vectors derived from naturally occurring human adeno-associated viruses and the GeneRide technology. LogicBio designed its approach to harness the natural power of homologous recombination − enabling precise, site-specific transfer of the genetic material without the use of promoters or nucleases. The goal: repairing the faulty genetic sequence causing the disorder to deliver a cure. LogicBio is advancing programs targeting rare, life-threatening pediatric genetic diseases of the liver, where there are few, if any, treatment options.

TECHNOLOGY

GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV-based gene therapies.
With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types. The company’s technology hitches a therapeutic transgene to a native promoter without disrupting its normal function in the host genome, enabling permanent expression of a functional version of a missing or faulty gene. Thus − with just a single treatment early in a child’s life – LogicBio can potentially deliver a life-long cure from a devastating disease.

1) The GeneRide technology harnesses the natural power of homologous recombination to enable precise, site-specific transfer of therapeutic genetic material without the use of promoters or nucleases. This strategy minimizes the risk of carcinogenicity spurred on by strong promoters used in traditional gene-therapy approaches.
2) A library of novel synthetic,non-pathogenic recombinant adeno-associated viral (rAAV) vectors, designed specifically to enable significant clinical performance advantage over established clinical capsids.

Products and Phases

Product	Phase
LB-001	Phase I
LB-001 is an investigational, first-in-class, single-administration, genome editing therapy for early intervention in methylmalonic acidemia (MMA) using LogicBio®'s proprietary GeneRide® drug development platform. GeneRide technology utilizes a natural DNA repair process called homologous recombination that enables precise editing of the genome without the need for exogenous nucleases and promoters that have been associated with an increased risk of immune response and cancer. LB-001 is designed to non-disruptively insert a corrective copy of the methylmalonyl-CoA mutase (MMUT) gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver, the main site of MMUT expression and activity. LB-001 is delivered to hepatocytes intravenously via liver-targeted, engineered recombinant adeno-associated virus vector (rAAV-LK03). Preclinical studies found that LB-001 was safe and demonstrated transduction of hepatocytes, site-specific genomic integration, and transgene expression. LB-001–corrected hepatocytes in a mouse model of MMA demonstrated preferential survival and expansion (selective advantage), thus contributing to a progressive increase in hepatic MMUT expression over time. LB-001 resulted in improved growth, metabolic stability, and survival in MMA mice. The U.S. Food and Drug Administration (FDA) granted fast track designation, rare pediatric disease designation and orphan drug designation for LB-001 for the treatment of MMA. In addition, the European Medicines Agency (EMA) granted orphan drug designation for LB-001 for the treatment of MMA.
LB-101
Hemophilia B
LB-201
A1ATD
LB-301	Phase I
Crigler-Najjar

	TARGET MARKETS
	Patients who suffer from life-threatening genetic diseases, or with early-onset rare diseases and infectious diseases, including: hemophilia, HIV/AIDS and methylmalonic acidemia (MMA).

	BUSINESS MODEL
	B2C

REMARKS

Date	Remark
16/11/2022	The company was acquired by Alexion, a subsidiary of AstraZeneca. No Israeli presence following the acquisition.
01/11/2019	The company closed its operations in Israel.

CONTACT INFO

Expand / Collapse All

Main Address: Cambridge, United States

Address	610 Main St., North 3 rd Floor
City	Cambridge
State/Province	MA
Country	United States
Zip	02139

Contact Person	abC Defghij
Contact E-Mail	abC Defghij
Company E-mail	abC Defghij
Telephone	abC Defghij
Fax	abC Defghij

	MANAGEMENT

Key Executive

Name	Title
Dr. Mark Kay	Co-Founder
Prof. Adi Barzel	Co-Founder
Dr. Leszek Lisowski	Co-Founder

Mergers & Acquisitions

Round

Date

Stage

Amount

Valuation

Acquired

Nov. 16, 2022

R&D

abCDe

Public Offerings

Deal Date	Type of Offering	Exchange	Amount Raise	Valuation	Remarks
Oct. 21, 2018	IPO	NASDAQ GM	abCDe	abCDe	SBI Japan-Israel Innovation Fund holds shares of the company post IPO

	FINANCIAL ROUNDS

Total Capital Raised
abCDe

Financial Rounds

Numbers of Investors

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Round

Date

Stage

Amount

Valuation

Second Round

Jun. 28, 2017

R&D

abCDe

* New investor

Investor	Type	Amount	Remarks
abC Defghij	Venture Capital Fund	abCDe	Round co-leader
abC Defghij	Venture Capital Fund	abCDe
abC Defghij	Venture Capital Fund	abCDe
abC Defghij	Private Equity Fund	abCDe
abC Defghij	High-Tech Company	abCDe	Round co-leader

First Round

Jun. 20, 2016

R&D

abCDe

Last Updated:	abCDe
IVC Number:	abCDe