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            Virtual BoothGlobal Pharmaceutical Dosage Solutions Company

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            Lead Product(s): Allogeneic cardiosphere-derived cells

            Therapeutic Area: Genetic Disease Product Name: CAP-1002

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Capricor Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration January 12, 2021

            Details:

            Capricor to leverage Lonza’s expertise in technology transfer and development of cellular therapies to take CAP-1002 through potential product launch and commercial supply.

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            Lead Product(s): Vamorolone

            Therapeutic Area: Genetic Disease Product Name: VBP15

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Highbridge Capital Management

            Deal Size: $16.5 million Upfront Cash: Undisclosed

            Deal Type: Financing November 02, 2020

            Details:

            Vamorolone, the only dissociative steroid in development, is currently in a pivotal Phase 2b study in DMD patients with a 6-month readout expected in the second quarter of 2021.

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            Lead Product(s): Vamorolone

            Therapeutic Area: Genetic Disease Product Name: VBP15

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: ReveraGen BioPharma

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 14, 2020

            Details:

            Data further show that vamorolone uniformly weakens co-activator associations, which leads to loss of gene transcriptional activities associated with safety concerns of corticosteroids.

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            Lead Product(s): Vamorolone

            Therapeutic Area: Genetic Disease Product Name: VBP15

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Santhera Pharmaceuticals

            Deal Size: $11.4 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 02, 2020

            Details:

            Santhera has obtained an exclusive license from ReveraGen, the originator of vamorolone, for all indications worldwide. Vamorolone is currently being investigated in the pivotal Phase 2b VISION-DMD study in patients with Duchenne muscular dystrophy (DMD).

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            Lead Product(s): ER-004

            Therapeutic Area: Genetic Disease Product Name: ER-004

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 06, 2020

            Details:

            The Breakthrough Therapy Designation was granted following the promising results from three XLHED subjects who were treated by Professor Holm Schneider with a course of ER-004 intra-amniotic injections during the third trimester of pregnancy.

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            Lead Product(s): Vamorolone

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 02, 2020

            Details:

            Data from this VBP15-003 study in comparison to natural history study data demonstrated dose-dependent improvement in timed function tests. Vamorolone was reported to be safe and well tolerated up to the highest dose tested (6.0 mg/kg/day).