HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based gene-editing tools and gene therapies to rewrite the future of genomic medicine. The robust pipeline covering CNS, ophthalmology, myology, otology, hepatology and novel technologies include:CNS:- HG204: RNA editing (hfCas13Y) MECP2 duplication syndrome- HG2A2019: DNA editing (Cas12) Huntington's disease- HG2A2103: DNA editing (Cas12) ALS- other discovery CNS programs in Parkinson's disease, Angelman syndrome, Spinocerebellar ataxia type 3 etc.Ophthalmology:- HG004: AAV gene replacement RPE65-mediated inherited retinal dystrophies- HG202: RNA editing (hfCas13Y) neovascular age-related macular degeneration- other discovery ocular programs in ABCA4-mediated Stargardt disease, corneal dystrophies, etc.Otology:-HG205: RNA base-editing (mxABE) OTOF-associated hearing lossMyology:-HG302: DNA editing (Cas12) Duchenne muscular dystrophyHepatology:-Transthyretin amyloidosis (ATTR) -HBV

A global clinical-stage biotech company focusing on discovering, engineering, and developing CRISPR-based gene therapy to rewrite the future of genome medicine. Product pipeline includes RPE65-associated inherited retinal dystrophies, neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy, MECP2 duplication syndrome.

-Global drug development strategist with more than 30 years of biotech/pharmaceutical experience in multi-portfolio management (antibodies, protein biologics, biosimilars, and gene therapy) to define strategic planning and pipeline growth.-Former member at the industrial board of the US FDA Rare Disease Clinical Design Committee.-Key core members to win regulatory marketing approvals of 21 products in gene therapy, neurology, ophthalmology, hematology, and cancer therapy as of today.-Versatile problem-solver with over 24 years of experience in program/project management.-Decisive leader with at least 21 years of experience in global clinical development operations and global medical affairs championing multiple products from First-in-Human study to marketing approval/launch.-Relationship-builder with >20 years of experience in KOL / Patient Advocacy / Consortia / Society management noted for preserving ties to clinical and scientific leaders and organizations.-Passionate mentor with 20+ years' line management /personnel development experience.Therapeutic Focus:- Hematology: DVT/VTE; Hemophilia A; Hemophilia B; Hemostasis; Hepatoma; VWF.- Oncology: Acute Myeloid Leukemia; Metastatic Breast Cancer, Metastatic Colorectal Cancer; Gastroesophageal Cancer; hepatocellular carcinoma; Glioma; Ovarian Cancer.- Neurology: Addiction; Alcoholism; Alzheimer's; Depression; Huntington's; Lysosomal Storage Disorder; Neuropathic Pain; Parkinson's; MDS, RETT, ALS.- Ophthalmology: Age-related Macular Degeneration (wet/dry); Choroideremia; Dominant Optic Atrophy; Leber Congenital Amaurosis; LHON; Retinitis Pigmentosa.- Metabolism: Hyperlipidemia; Liver Disorders; Obesity; Type II Diabetes.

- Namenda-XR- Vivitrol- Campral- Herceptin- Lucentis

Our mission is to discover and develop AAV2 gene therapies for ocular diseases.

Developing AAV-gene therapy product candidates for the treatment of genetic ocular diseases.

A global role that is responsible for delivering strategic clinical programs, driving operational efficiencies, implementing business change and expanding R&D portfolio by in-/out-licensing evaluations.- HLX10 in sNSCLC- HLX10 in SCLC- HLX10 + HLX04 (bevacizumab biosimilar) in HCC- HLX04 (bevacizumab biosimilar) for wAMD and DME

A. Biosimilars (Oncology and Immunology) - rituximab (approved on 2/2019 in China; 汉利康® /HanLiKang) - trastuzumab (approved on 8/2020 in China - 汉曲优®, approved on 7/2020 in EU - Zercepac®, and approved on 4/2024 in US - HERCESSI™) - adalimumab (approved on 12/2020 in China - 汉达远® /HanDaYuan) - bevacizumab (approved on 11/2022 in China - 汉贝泰® /HanBeiTai) - cetuximab (Phase 1b/2 trial is ongoing in China) - pertuzumab (Phase 3 trial is ongoing in China) - ramucirumab (Phase 1 trial is ongoing in China) - ipilimumab (IND enabling) - denosumab (Phase 3 trial is ongoing in China) - daratumumab (Phase 1 trial is ongoing in China) - pembrolizumab (IND enabling) - nivolumab (IND enabling)B. Innovative monoclonal antibodies (Oncology and Infection): - anti-EGFR (Phase 2 trials are ongoing in P.R. China and Taiwan) - anti-VEGFR2 (FiH trial is ongoing in Taiwan) - anti-CTLA-4 (IND enabling) - anti-PD-1 (approved on 3/2022 in China - 汉斯状® /HanSiZhuang, Serplulimab) - anti-PD-L1 (FiH trial is ongoing in Australia) - anti-c-Met (Phase 1 trial is ongoing in China and Taiwan) - anti-LAG3 (Phase 1 trial is ongoing in China) - anti-death-receptor (IND enabling) - anti-S1 of SARS-CoV-2 (Phase 1 trial is ongoing in China)receptor binding domain (RBD) of S1 subunit of S protein on the surface of the virus C. First-/Best-in-Class (Hematology, Oncology, Immunology and Rare Diseases) - Bispecific (IND enabling) - Cell (CAR-T) and Gene therapies (clinical trials in China are ongoing)

- AAV Gene Therapy for Rare Genetic Diseases- SPK9001 - Hemophilia B -collaboration with Pfizer (BEQVEZ)- SPK8011 - Hemophilia A- SPK-7001 - Choroideremia- Batten (CLN-2) Disease- Huntington's Disease

Global Medical Affairs- Eloctate (recombinant factor VIII Fc-fusion protein for patients with hemophilia A: from BLA submission to FDA approval and launch)- Alprolix (recombinant factor IX Fc-fusion protein for patients with hemophilia B: from BLA submission to FDA approval and launch)Global Clinical Development Operations-Recombinant factor VIII Fc-fusion protein for hemophilia A (from PoC clinical trial to BLA submission)-Recombinant factor IX Fc-fusion protein for hemophilia B (from PoC clinical trial to BLA submission)

FDA-Rare disease clinical design committee: Focusing on optimizing clinical trial design for rare diseases to shorten drug development timeline

- Extended half-life coagulation factor VIII product development: a. Liposomal-BAY-79-4980 b. PEGylated-BAY-94-9027, Jivi- Life-cycle management of Kogenate (expanded label indication of Kogenate-FS for "children prophylaxis" and BAY 81-8973, Kovaltry)- Supports in China and South East Asia Kogenate-FS launches- In-licensing review of recombinant Thrombin and factor VIIa candidates

- Adeno-associated vector serotype 2 (AAV2) encoding human coagulation factor IX (FIX) for hemophilia B- Adeno-associated vector serotype 2 (AAV2) encoding human aromatic L-amino acid decarboxylase (AADC) for parkinson disease- AV-411 for neuropathic pain

Identifying lead candidates using LxxLL-motif agonist and antagonist of orphan nuclear receptors screening which acted through the regulation of gene expression:a. PPARa,b,d and -g (peroxisome proliferator-activated receptors)b. ERRa, b and -g (estrogen-related receptors)c. RXRa, b and -g (9-cis retinoic acid receptors)d. NURR1 (nuclear receptor related-1 protein)e. LXRa and -b (liver X receptors) f. FXR (farnesoid X receptor: discovery of natural antagonist)