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Cell & Gene Therapy Center of Excellence

Connecting patients with revolutionary cell and gene therapies

Orsini’s Cell and Gene Therapy Center of Excellence launched in 2017 with our first cell therapy product for cartilage defect repair. In 2019, we proudly added our first gene therapy product for spinal muscular atrophy. Today, we support five programs across rare conditions in hematology, neurology, and orthopedics.

Cell and gene therapies represent cutting-edge medical approaches with immense promise for treating rare diseases. Cell therapy involves the infusion or transplantation of living cells, often engineered or modified, into a patient’s body to restore or enhance their function. Gene therapies alter faulty genes or replace them with healthy ones to correct genetic disorders at the molecular level. These innovative treatments offer targeted and potentially curative solutions for rare diseases by addressing the underlying causes rather than just alleviating symptoms. By harnessing the power of genetics and cellular biology, cell and gene therapies pave the way for personalized medicine, providing hope to patients with previously untreatable conditions.

Explore Our Cell and Gene Therapies

Benefits

Improving the Patient Experience, Every Time

Single Point of Contact

Patients, caregivers, prescribers and other members of the care team work with a single point of contact at Orsini who supports every step of the way and is available via phone, email, and fax.

Dedicated Care Team

Receive personalized support from a dedicated Cell and Gene Therapy Care Team, including a primary pharmacy contact, clinical education, side-effects management, and scheduled refill calls.

Coordinated and Timely Care

On average, we can onboard any new cell and gene patient in 5-7 business days and schedule shipment to the appropriate facility.

Insurance Coverage

Orsini has established national contracts with commercial payors and Medicaid, ensuring that patients have access to the therapies they need.

Financial Assistance

Minimize financial exposure through financial assistance programs. We successfully identify and assist in enrolling patients into manufacturer co-pay and foundation support programs.

24/7/365 Access to a Pharmacist

Gain continuous access to a pharmacist, 24/7/365, ensuring ongoing care and clinical support for patients and prescribers to achieve better outcomes.

Support

Cell and Gene Therapies Supported

Explore the rare and complex conditions Orsini supports with cell and gene therapy programs below.

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

AADC deficiency is a rare and fatal genetic disorder that can cause severe physical, mental, and behavioral disability and suffering starting in infancy. Those suffering from AADC deficiency may also experience episodes that resembling seizures called oculogyric crises and deal with complications such as infections and severe feeding and breathing problems.

AADC Deficiency Therapies Supported

 

Full Name KEBILIDI™ (eladocagene exuparvovec-tneq)
Drug Kebilidi
Manufacturer PTC Therapeutics, Inc.
Route of Administration Intraputaminal
Site of Care Healthcare facility
Approved Indication Indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency
Disease Aromatic L-amino acid decarboxylase (AADC) deficiency
Therapeutic Area Neurology, Genetics, Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 888-221-3495
Fax Number 877-810-7806
Product Website kebilidi.com

About Cartilage Defect Repair

Cartilage is the tough but flexible tissue that covers the ends of your bones at a joint. Healthy cartilage helps people move by allowing the bones to glide over each other. It also protects bones by preventing them from rubbing against each other. Unlike other tissues, cartilage does not repair itself and can become a chronic problem, often getting worse as time goes on. If conservative treatment does not work to relieve pain, surgery is often the answer.

Cartilage Defect Repair Therapies Supported

Full Name MACI® (autologous cultured chondrocytes on a porcine collagen membrane)
Drug MACI
Manufacturer Vericel Corporation
Route of Administration Surgery
Site of Care Healthcare Facility
Approved Indication The repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults
Disease Cartilage Defect Repair
Therapeutic Area Orthopedics; Cell & Gene Therapy
Enrollment Form Link MACI Enrollment Form
Phone Number 800-388-1903
Fax Number N/A
Product Website maci.com

About Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD) is a severe form of X‑linked adrenoleukodystrophy (X‑ALD), a genetic disorder caused by mutations in the ABCD1 gene. The mutation impairs the body’s ability to break down very long‑chain fatty acids (VLCFAs), which accumulate, leading to damage the nerve cells in the brain. CALD typically appears in boys between ages 4 and 10 and may cause learning or behavioral changes, difficulty concentrating, and coordination problems. As the disease advances, it can lead to loss of vision, hearing, mobility, cognitive functions, and death within a few years of symptom onset.

CALD Therapies Supported

 

Full Name SKYSONA® (elivaldogene autotemcel)
Drug elivaldogene autotemcel
Manufacturer Genetix Biotherapeutics Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant
Disease Cerebral Adrenoleukodystrophy (CALD)
Therapeutic Area Neurology; Cell & Gene Therapy
Enrollment Information Visit skysona.com
Phone Number 847-849-1588
Fax Number 877-860-1726
Website skysona.com

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy is a genetic, progressive form of muscular dystrophy that occurs primarily in males. It is caused by genetic changes in the DMD gene and is inherited in an X-linked recessive pattern. X-linked means the gene for the condition is located on the X-chromosome. In males, one altered copy of the gene is enough to cause DMD. DMD causes progressive weakness and muscle atrophy. In early signs of the disease, DMD may cause a delayed ability to sit, stand, or walk, and can make learning to speak difficult for the patient.

DMD Therapies Supported

Full Name Elevidys™ (delandistrogene moxeparvovec-rokl)
Drug Elevidys
Manufacturer Sarepta Therapeutics, Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age:
  • For patients who are ambulatory and have a confirmed mutation in the DMD gene
  • For patients who are non-ambulatory and have a confirmed mutation in the DMD gene
Disease Duchenne muscular dystrophy (DMD)
Therapeutic Area Neurology; Genetics; Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 800-870-8931
Fax Number 877-694-2546
Product Website elevidyshcp.com
Patient Resources Alex Rezcalla Foundation, Jett Foundation, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy

About Hemophilia A

Hemophilia A, also called factor VIII deficiency or classic Hemophilia, is an X-linked genetic disorder caused by missing or defective factor VIII, a clotting protein. Approximately 1 in 10,000 people have Hemophilia A. Individuals with the most severe form of hemophilia A make up approximately 50% of the Hemophilia A population.

Hemophilia A Disease Therapies Supported

Full Name Roctavian™ (valoctocogene roxaparvovec-rvox)
Drug Roctavian
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test
Disease Hemophilia A
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 800-358-0013
Fax Number 877-631-9228
Product Website roctavian.com

About Hemophilia B

Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births. People with this condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Hemophilia B can range from mild (factor IX levels between 5 and 40% of normal) to moderate (factor IX levels from 1 to 5% of normal) to severe (factor IX levels less than 1% of normal).

Hemophilia B Therapies Supported

Full Name Hemgenix® (etranacogene dezaparvovec-drlb)
Drug Hemgenix
Manufacturer CSL Behring
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes
Disease Hemophilia B
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 800-975-8693
Fax Number 877-740-7535
Product Website hemgenix.com/hcp

About Idiopathic Macular Telangiectasia (MacTel) Type 2

Idiopathic macular telangiectasia (MacTel) type 2 is a rare, progressive eye disease that affects the part of the retina responsible for central vision. It typically occurs in both eyes and leads to gradual vision loss, often beginning with subtle symptoms like blurred or distorted vision. While the exact cause is unknown, genetic factors and systemic conditions like diabetes may contribute to its development.

MacTel Type 2 Therapies Supported

 

Full Name ENCELTO™ (revakinagene taroretcel-lwey)
Drug Encelto
Manufacturer Neurotech Pharmaceuticals, Inc.
Route of Administration Intravitreal
Site of Care Healthcare facility
Approved Indication Indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel)
Disease Idiopathic macular telangiectasia type 2 (MacTel)
Therapeutic Area Ophthalmology; Cell & Gene Therapy
Enrollment Form Link Start Form
Phone Number 888-477-1165
Product Website ENCELTO.com/ecp

About Sickle Cell Disease

Sickle cell disease is an inherited blood disorder causing red blood cells to become “sickled,” leading to severe pain, organ damage, and shorter life expectancy. Hallmarked by painful blood vessel blockages (vaso-occlusive crises, or VOCs), SCD can also cause acute chest syndrome, stroke, jaundice, and heart failure symptoms.

Sickle Cell Disease Therapies Supported

 

Full Name LYFGENIA™ (lovotibeglogene autotemcel)
Drug lovotibeglogene autotemcel
Manufacturer Genetix Biotherapeutics Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events
Disease Sickle Cell Disease (SCD)
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Information Visit lyfgeniahcp.com
Phone Number 847-849-1588
Fax Number 877-860-1726
Website lyfgeniahcp.com

Full Name CASGEVY™ (exagamglogene autotemcel)
Drug Casgevy
Manufacturer Vertex Pharmaceuticals
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of patients aged 12 years and older with:
  • Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
  • Transfusion-dependent β-thalassemia (TDT)
Disease Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT)
Therapeutic Area Hematology; Cell & Gene Therapy
Phone Number 1-833-837-8395

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a genetic disease that affects the central nervous system, peripheral nervous system, and voluntary muscle movement, also known as skeletal muscle. A majority of the nerve cells that control muscles are located in the spinal cord, and when the patient’s muscles do not receive signals from those nerve cells, this results in their muscles getting smaller. The cause of spinal muscular atrophy is due to a missing gene that encodes the survival motor neuron (SMN) protein, which is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons). SMA occurs in nearly 1 in 11,000 births in the United States. The age when SMA symptoms begin tends to correlate with the severity to which motor function is affected.

SMA Therapies Supported

Full Name Zolgensma® (onasemnogene abeparvovec-xioi)
Drug Zolgensma
Manufacturer Novartis Gene Therapy
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene
Disease Spinal Muscular Atrophy (SMA)
Therapeutic Area Neurology; Cell & Gene Therapy
Enrollment Form Enrollment Form
Phone Number 800-697-5048
Fax Number 877-471-5704
Website zolgensma.com
Patient Resources Cure SMA and Muscular Dystrophy Association

Full Name ITVISMA® (onasemnogene abeparvovec-brve)
Drug onasemnogene abeparvovec-brve
Manufacturer Novartis Gene Therapy
Route of Administration Intrathecal
Site of Care Healthcare Facility
Approved Indication The treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene
Disease Spinal Muscular Atrophy (SMA)
Therapeutic Area Neurology; Cell & Gene Therapy
Enrollment Form Enrollment Form
Phone Number 800-697-5048
Fax Number 877-471-5704
Website itvisma.com
Patient Resources Cure SMA and Muscular Dystrophy Association

About Transfusion-Dependent Beta Thalassemia (TDT)

Transfusion-dependent beta thalassemia is an inherited blood disorder causing anemia, resulting in fatigue, shortness of breath, and, in infants, growth issues and jaundice. Complications can include an enlarged spleen, liver, or heart; misshapen bones; and delayed puberty. Many patients need regular blood transfusions, requiring frequent hospital visits and often causing iron buildup.

TDT Therapies Supported

 

Full Name ZYNTEGLO™ (betibeglogene autotemcel)
Drug betibeglogene autotemcel
Manufacturer Genetix Biotherapeutics Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions
Disease Transfusion-dependent β-thalassemia (TDT)
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Information Visit zynteglohcp.com
Phone Number 847-849-1588
Fax Number 877-860-1726
Website zynteglohcp.com

Full Name CASGEVY™ (exagamglogene autotemcel)
Drug Casgevy
Manufacturer Vertex Pharmaceuticals
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of patients aged 12 years and older with:
  • Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
  • Transfusion-dependent β-thalassemia (TDT)
Disease Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT)
Therapeutic Area Hematology; Cell & Gene Therapy
Phone Number 1-833-837-8395

Patient Advocacy Partners