Bill Aliski

We are a commercial-stage clinical biopharmaceutical company driven to improve the lives of people with rare diseases of the immune system and few to no treatment options. We have successfully developed mavorixafor, receiving FDA approval for XOLREMDI® (mavorixafor) in its first indication, and are currently executing our US launch. We are also advancing mavorixafor for additional rare disease patient populations, and expect to initiate a pivotal, global Phase 3 clinical trial in people with certain chronic neutropenic disorders. With a focus on CXCR4 and immune system biology, we continue to leverage our expertise both at our corporate headquarters in Boston, Massachusetts and at our research center of excellence in Vienna, Austria to further advance our patient-centric mission.

Scioderm, Inc. was acquired by Amicus Therapeutics, Inc. in September of 2015. Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering high-quality medicines for people living with rare genetic diseases. Amicus, together with Scioderm, was developing an investigational topical treatment (SD-101) for people with Epidermolysis Bullosa (EB), which is a rare genetic connective tissue disorder. In September of 2017, results of a Phase 3 clinical study to assess the efficacy and safety of SD-101, showed that it did not show a statistically significant difference from placebo. Subsequently, Amicus discontinued the program, but shared the clinical data and additional information related to the EB program with key stakeholders including other companies with investigational treatments for EB as well as the broader EB community.

Edimer Pharmaceuticals: Working toward improving the health and quality of life of families living with XLHED. Edimer Pharmaceuticals is dedicated to developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED). XLHED is a rare, orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations. Edimer is led by a team of seasoned biotechnology industry veterans with deep experience in drug development complemented by a strong network of world-class clinical and scientific advisers. The company is based in Cambridge, Massachusetts.

We are a commercial-stage clinical biopharmaceutical company driven to improve the lives of people with rare diseases of the immune system and few to no treatment options. We have successfully developed mavorixafor, receiving FDA approval for XOLREMDI® (mavorixafor) in its first indication, and are currently executing our US launch. We are also advancing mavorixafor for additional rare disease patient populations, and expect to initiate a pivotal, global Phase 3 clinical trial in people with certain chronic neutropenic disorders. With a focus on CXCR4 and immune system biology, we continue to leverage our expertise both at our corporate headquarters in Boston, Massachusetts and at our research center of excellence in Vienna, Austria to further advance our patient-centric mission.

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products Find AGTC on YouTube at goo.gl/sMdJqX.

Scioderm, Inc. was acquired by Amicus Therapeutics, Inc. in September of 2015. Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering high-quality medicines for people living with rare genetic diseases. Amicus, together with Scioderm, was developing an investigational topical treatment (SD-101) for people with Epidermolysis Bullosa (EB), which is a rare genetic connective tissue disorder. In September of 2017, results of a Phase 3 clinical study to assess the efficacy and safety of SD-101, showed that it did not show a statistically significant difference from placebo. Subsequently, Amicus discontinued the program, but shared the clinical data and additional information related to the EB program with key stakeholders including other companies with investigational treatments for EB as well as the broader EB community.

Edimer Pharmaceuticals: Working toward improving the health and quality of life of families living with XLHED. Edimer Pharmaceuticals is dedicated to developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED). XLHED is a rare, orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations. Edimer is led by a team of seasoned biotechnology industry veterans with deep experience in drug development complemented by a strong network of world-class clinical and scientific advisers. The company is based in Cambridge, Massachusetts.