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Research Team ACF

Regenerative Healthcare - Stem Cells & ALS Thematic 10062024

Regenerative medicine remains an extremely exciting area of healthcare with vast investment potential. In spite of the usual setbacks in valuation and strategy, the sub-sector has pivoted and the market remains innovative and attracts investment capital. Rather than replace entire tissues, which is a complex activity, we believe the immediate future for stem cells is more likely to be in the successful stimulation of the bodies on repair mechanisms. Within regenerative medicine we believe there is significant potential for stem cell therapies for neurodegenerative diseases. To illustrate we look at Motor Neuron Disease (MND) also called ALS or Lou Gehrig’s disease. Lou Gehrig was a baseball player, thought to be the first formally recognized sufferer of MND. We examine results from BCLI’s recent biomarker peer reviewed research in relation to the treatment of MND using a stem cell MSC-NTF proprietary platform called NurOwn (debamestrocel, MSC-NTF).
Underlyings
AbbVie Inc.

AbbVie is a research-based biopharmaceutical company. The company's products are focused on treating conditions such as chronic autoimmune diseases in rheumatology, gastroenterology and dermatology; oncology, including blood cancers; virology, including hepatitis C virus and human immunodeficiency virus; neurological disorders, such as Parkinson's disease; metabolic diseases, including thyroid disease and complications associated with cystic fibrosis; pain associated with endometriosis; as well as other serious health conditions. The company also has medicines in clinical development across immunology, oncology and neuroscience, with additional targeted investment in cystic fibrosis and women's health.

Amneal Pharmaceuticals Inc. Class A

Amneal Pharmaceuticals is a holding companies. Through its subsidiaries, the company is a pharmaceutical company enganged in developing, manufacturing, marketing and distributing pharmaceutical products in a variety of dosage forms and therapeutic categories. The company's pharmaceutical research includes analytical and formulation development and stability. The company has two business segments: Generics, which products are sold either as branded or generic products; and Specialty, which is engaged in the development, promotion, sale and distribution of proprietary branded pharmaceutical products, with a focus on products addressing central nervous system disorders, including migraine and Parkinson's disease.

Biogen Inc.

Biogen is a biopharmaceutical company focused on discovering, developing and delivering therapies for people living with neurological and neurodegenerative diseases as well as related therapeutic adjacencies. The company's main growth areas include multiple sclerosis (MS) and neuroimmunology; Alzheimer's disease and dementia; neuromuscular disorders, including spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis; movement disorders, including Parkinson's disease; and ophthalmology. The company's marketed products include TECFIDERA, AVONEX, PLEGRIDY, TYSABRI, VUMERITY and FAMPYRA for the treatment of MS, SPINRAZA for the treatment of SMA and FUMADERM for the treatment of severe plaque psoriasis.

Brainstorm Cell Therapeutics

Brainstorm Cell Therapeutics is a biotechnology company engaged in the development and commercialization of autologous cellular therapies for the treatment of neurodegenerative diseases including: Amyotrophic Lateral Sclerosis; Progressive Multiple Sclerosis; and Parkinson's disease. NurOwn? technology is based on manufacturing protocol, which induces the differentiation of purified and expanded bone marrow-derived mesenchymal stem cells and consistently generates cells that release multiple neurotrophic factors to modulate neuroinflammatory and neurodegenerative disease processes, promote neuronal survival and improve neurological function.

Bristol-Myers Squibb Company

Bristol-Myers Squibb is engaged in the discovery, development, licensing, manufacturing, marketing, distribution and sale of biopharmaceutical products. The company's products are sold worldwide, primarily to wholesalers, specialty distributors, retail pharmacies, hospitals, government entities and the medical profession. The company manufactures products in the United States and Puerto Rico and has manufacturing operations in two foreign countries. The company has products in the following therapeutic classes: hematology, oncology, cardiovascular and immunology. The company's pharmaceutical products include chemically-synthesized or small molecule drugs and products produced from biological processes, called biologics.

Celgene Corporation

Celgene is a global biopharmaceutical company engaged primarily in the discovery, development and commercialization of therapies for the treatment of cancer and inflammatory diseases through solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. The company's primary commercial stage products include: REVLIMID?, an oral immunomodulatory drug; POMALYST?/IMNOVID?, a proprietary, small molecule; OTEZLA?, an oral small-molecule inhibitor of phosphodiesterase 4 for cyclic adenosine monophosphate; ABRAXANE?, a solvent-free chemotherapy product; and VIDAZA?, a pyrimidine nucleoside analog. In addition, the company provides other product sales and licensing arrangements.

Cytokinetics Incorporated

Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing muscle activators and muscle inhibitors as treatments for debilitating diseases in which muscle performance is compromised and/or declining. The company's clinical-stage drug candidates are: omecamtiv mecarbil, a cardiac myosin activator for the treatment of heart failure, AMG 594, a cardiac troponin activator, reldesemtiv, a skeletal muscle troponin activator for the treatments of spinal muscular atrophy and amyotrophic lateral sclerosis, and CK-3773274, a cardiac myosin inhibitor which is designed to reduce the hypercontractility that is associated with hypertrophic cardiomyopathy.

Eisai Co. Ltd.

Eisai is mainly engaged in the manufacture and sale of pharmaceutical products. Co.'s principal prescription drugs include anticancer agents, anti-Alzheimer's agent, anti-TNF monoclonal antibodies, antiepileptic agents, proton pump inhibitors, neuropathic pain relievers, and other prescription drugs under the name of "Halaven," "Lenvima," "Fycompa," "BELVIQ," "Pariet/AcipHex," "Aricept," and "Humira." Co.'s principal over-the-counter products include Vitamin B2 preparations "Chocola BB," gastrointestinal drugs, anti-allergic drugs, and dermatological preparations. Co. operates its operations in Japan, the Americas, Europe, China and Asia.

Gilead Sciences Inc.

Gilead Sciences is a research-based biopharmaceutical company that discovers, develops and commercializes medicines. The company's primary areas of focus include viral diseases, inflammatory and fibrotic diseases and oncology. The company's products include: Biktarvy for the treatment of HIV-1 infection in certain patients; Vosevi?, a single tablet regimen of sofosbuvir, velpatasvir and voxilaprevir for the re-treatment of chronic hepatitis C virus infection in adults; Vemlidy? for the treatment of chronic hepatitis B virus infection in adults with compensated liver disease; and Yescarta (axicabtagene ciloleucel) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Ionis Pharmaceuticals Inc.

Ionis Pharmaceuticals is engaged in discovering and developing RNA-targeted therapeutics. The company has two commercial medicines, SPINRAZA and TEGSEDI. SPINRAZA (nusinersen) injection for intrathecal use is a survival motor neuron-2, directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy in pediatric and adult patients. TEGSEDI (inotersen) injection is a Generation 2+ antisense medicine and an approved subcutaneous RNA-targeting medicine designed to treat people with polyneuropathy caused by hereditary transthyretin amyloidosis.

Johnson & Johnson

Johnson & Johnson is a holding company engaged in the research and development, manufacture and sale of a range of products in the health care field. The company has three business segments: Consumer, which includes a range of products focused on personal healthcare used in the beauty, over-the-counter pharmaceutical, baby care, oral care, women's health and wound care markets; Pharmaceutical, which is focused on six therapeutic areas: immunology, infectious diseases, neuroscience, oncology, cardiovascular and metabolism and pulmonary hypertension; and Medical Devices, which includes products used in the orthopaedic, surgery, interventional solutions, and eye health fields.

Merck & Co. Inc.

Merck & Co. is a global health care company that delivers health solutions through its prescription medicines, vaccines, biologic therapies and animal health products. The company's segments are: Pharmaceutical, which includes human health pharmaceutical products that consist of therapeutic and preventive agents for the treatment of human disorders, and human health vaccine products that consist of preventive pediatric, adolescent and adult vaccines; and Animal Health, which discovers, develops, manufactures and markets veterinary pharmaceuticals, vaccines and health management solutions and services, as well as a suite of digitally connected identification, traceability and monitoring products.

Novartis AG

Novartis is a multinational healthcare group based in Switzerland. Co. provides healthcare solutions that address the evolving needs of patients and societies worldwide. Co.'s broad portfolio includes innovative medicines, eye care products and cost-saving generic pharmaceuticals. Co.'s operations are organized along three operating divisions: Innovative Medicines Division; Sandoz Division; and Alcon Division. Co.'s operations are supported by the Novartis Institutes for BioMedical Research and Novartis Business Services.

Pfizer Inc.

Pfizer is a research-based biopharmaceutical company. The company is engaged in discovering, developing, manufacturing and distributing of healthcare products, including medicines and vaccines. The company manages its commercial operations through three businesses: Pfizer Biopharmaceuticals Group, which includes Oncology, Inflammation and Immunology, Rare Disease, Hospital, Vaccines and Internal Medicine business units, as well as a hospital business unit; Upjohn, which includes the company's solid oral dose brands such as Lyrica, Lipitor, Norvasc, Celebrex, Viagra, and certain generic medicines; and Consumer Healthcare, which is an over-the-counter medicines business.

Roche Holding Ltd

Roche Holding is a healthcare company. Co. operates in two Divisions, Pharmaceuticals and Diagnostics. Co.'s pharmaceuticals division is comprised of two business segments Roche Pharmaceuticals and Chugai. Co.'s pharmaceuticals division provides therapies including Cotellic, in combination with Zelboraf for advanced melanoma, and Alecensa for a specific type of lung cancer. Co.'s diagnostics division consists of four business areas: Diabetes Care, Molecular Diagnostics, Professional Diagnostics and Tissue Diagnostics. Co.'s diagnostics division provides in vitro diagnostic tests, used by healthcare providers to screen, diagnose and monitor therapies.

Sanofi

Sanofi is an integrated, global healthcare company focused on patient needs and engaged in the research, development, manufacture and marketing of healthcare products. Two segments make up Co.'s business: Pharmaceuticals and Vaccines. The Pharmaceuticals segment covers research, development, production and marketing of medicines, consisting of flagship products, plus a broad range of prescription medicines, generic medicines, and consumer health products. The Vaccines segment includes research, development, production and marketing of vaccines in five areas: pediatric vaccines, influenza vaccines, adult and adolescent booster vaccines, meningitis vaccines, and travel and endemic vaccines.

Teva Pharmaceutical Industries Limited

Teva Pharmaceutical Industries is engaged in the development, manufacturing, marketing and distribution of generic, specialty, and other pharmaceutical products. The Generic Medicines segment offers a range of basic chemical entities, as well as specialized product families such as sterile products, hormones, narcotics, high-potency drugs and cytotoxic substances, in both parenteral and solid dosage forms. Co.'s specialty medicines business is focused on delivering innovative solutions to patients and providers via medicines, devices and services, includes Co.'s core therapeutic areas of the central nervous system and respiratory medicines.

UNIQURE NV

uniQure is a biopharmaceutical company. Co. is engaged in the field of gene therapy, seeking to develop single treatments with potentially curative results for patients suffering from genetic and other diseases. Co. is advancing a focused pipeline of gene therapies that have been developed both internally and through partnerships, such as Co.'s collaboration with Bristol Myers-Squibb focused on cardiovascular diseases. Co. has established clinical proof-of-concept in its key indication, hemophilia B, and achieved preclinical proof-of-concept in Huntington's disease.

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