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01

Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : The agreement aims to commercialize Libmeldy (atidarsagene autotemcel), the only approved therapy for eligible children with early-onset metachromatic leukodystrophy (MLD).

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Undisclosed

July 10, 2024

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Genetic Disease

Highest Development Status : Approved FDF

Sponsor : Erkim

Deal Size : Undisclosed

Deal Type : Partnership

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02

Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : OTL-203 is an investigational hematopoietic stem cell gene therapy using a modified virus to insert a functional IDUA gene copy into patient cells, under Phase 3 for MPS-I Hurler Syndrome.

Product Name : OTL-203

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

May 02, 2024

Lead Product(s) : OTL-203

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : San Raffaele-Telethon Institute for Gene Therapy

Deal Size : Inapplicable

Deal Type : Inapplicable

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03

Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion of CD34+ cells containing hematopoietic stem cells, approved for early-onset metachromatic leukodystrophy treatment.

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

March 20, 2024

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Inapplicable

Deal Size : Inapplicable

Deal Type : Inapplicable

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04

Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion of CD34+ cells containing hematopoietic stem cells, approved for early-onset metachromatic leukodystrophy treatment.

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

March 18, 2024

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Inapplicable

Deal Size : Inapplicable

Deal Type : Inapplicable

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05

Pharma Live Expo
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Pharma Live Expo
Not Confirmed

Details : The agreement aims for enabling reimbursed access to Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for early-onset metachromatic leukodystrophy (MLD).

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Undisclosed

January 25, 2024

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Beneluxa Initiative

Deal Size : Undisclosed

Deal Type : Agreement

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Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : Kyowa expands its portfolio to develop candidates with a differentiated platform and supports ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : $387.4 million

January 24, 2024

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Kyowa Kirin

Deal Size : $477.6 million

Deal Type : Acquisition

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Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : OTL-203 is an IDUA gene Transference cell and gene therapy drug infused intravenously, it is being investigated for the treatment of Hurler subtype of mucopolysaccharidosis type I.

Product Name : OTL-203

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

November 30, 2023

Lead Product(s) : OTL-203

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Inapplicable

Deal Size : Inapplicable

Deal Type : Inapplicable

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Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : OTL-200 (libmeldy or atidarsagene autotemcel), is a hematopoietic stem cell (HSC) gene therapy approved by Swissmedic for the treatment of early-onset metachromatic leukodystrophy (MLD).

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

November 12, 2023

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Inapplicable

Deal Size : Inapplicable

Deal Type : Inapplicable

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Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enz...

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Inapplicable

September 18, 2023

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Approved FDF

Sponsor : Inapplicable

Deal Size : Inapplicable

Deal Type : Inapplicable

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Pharma Live Expo
Not Confirmed
Pharma Live Expo
Not Confirmed

Details : The Company intends to use the net proceeds to accelerate commercialization of Libmeldy (atidarsagene autotemcel), a gene therapy treatment for metachromatic leukodystrophy, and advance the company’s HSC gene therapy R&D portfolio and approval of OTL-2...

Product Name : Libmeldy

Product Type : Cell and Gene therapy

Upfront Cash : Undisclosed

June 26, 2023

Lead Product(s) : Atidarsagene Autotemcel

Therapeutic Area : Rare Diseases and Disorders

Highest Development Status : Phase III

Sponsor : Guggenheim Securities

Deal Size : $188.0 million

Deal Type : Private Placement

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