Substrate reduction therapy

Waldenström macroglobulinemia

Light therapy

Redox therapy

Feminizing hormone therapy

Phage therapy

Vintafolide

Sodium oxybate

Daprodustat

Baloxavir marboxil

Stem cell tourism

Ebselen

Cioteronel

List of investigational attention deficit hyperactivity disorder drugs

Tay-Sachs disease

Social history of viruses

Jason Emer

Primary myelofibrosis

Extracranial germ cell tumor

Preterm birth

Tinnitus masker

Esreboxetine

Niemann-Pick disease, type C

PIM1

Dinaciclib

List of investigational obsessive-compulsive disorder drugs

Psoriasis

Sensory stimulation therapy

Cutibacterium acnes

4-Ipomeanol

List of investigational sex-hormonal agents

Access to investigational therapies1

• Sometimes called "compassionate use", expanded access is a potential pathway for a patient to gain access to investigational therapies in certain rare circumstances in which a person has no other available therapies or is not eligible to participate in a current Eureka clinical trial. (eurekaconnectme.com)

Provide our investigational2

• As a general policy, Eureka will not provide our investigational therapies until sufficient preliminary safety and efficacy information has been obtained in our clinical trials, typically following Phase 2 investigation. (eurekaconnectme.com)
• We continue to be proud and humbled to provide our investigational V920 Ebola vaccine as an additional tool in support of the comprehensive public health response efforts against the current Ebola outbreak. (merck.com)

Therapeutics13

• PTC Therapeutics has presented new long-term outcome data from its investigational gene therapy, PTC-AADC, in patients living with aromatic L-amino acid decarboxylase (AADC) deficiency. (pharmatimes.com)
• Eureka Therapeutics is committed to developing next-generation T cell therapies for cancer patients who have serious and life-threatening diseases or conditions. (eurekaconnectme.com)
• NEW YORK and CLEVELAND, May 21, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company is cleared to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease, following acceptance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). (abeonatherapeutics.com)
• Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. (abeonatherapeutics.com)
• For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is the Buzz of BIO 2020 winner in the Public Therapeutic Biotech category. (alsnewstoday.com)
• SB-525 gene therapy, which is being developed as part of a global collaboration between Sangamo Therapeutics and Pfizer, sees positive growth following promising data from their phase 1/2 Alta study. (geneonline.com)
• On July 6, Sangamo Therapeutics, and Pfizer announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH), in Melbourne, Australia. (geneonline.com)
• The manufacture of potentially transformative gene therapy technologies is an incredibly complex undertaking, and we are hopeful that this key milestone further strengthens our relationship with Pfizer while ensuring the long-term development of VTX-801," said Jean-Philippe Combal, Co-Founder & CEO of Vivet Therapeutics. (vivet-therapeutics.com)
• Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases. (vivet-therapeutics.com)
• LEXEO Therapeutics is a New York City-based, clinical-stage gene therapy company focused on addressing some of the most devastating genetically defined cardiovascular and central nervous system diseases affecting both larger-rare and prevalent patient populations. (lexeotx.com)
• SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced that its Investigational New Drug (IND) application for its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN), was cleared by the U.S. Food and Drug Administration (FDA). (synconaltd.com)
• SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. (synconaltd.com)
• CAMBRIDGE, MA / ACCESSWIRE / May 30, 2023 / Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that two abstracts on mRNA-4157 (V940), an investigational mRNA individualized neoantigen therapy, have been accepted for presentation at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting being held June 2-6 in Chicago, IL. (malvern-online.com)

Company's4

• Stamford, Conn. - April 24, 2019 - Purdue Pharma L.P. today announced that the U.S. Food & Drug Administration ( FDA )'s Office of Generic Drugs has granted Competitive Generic Therapy ( CGT ) designation to nalmefene hydrochloride (HCl) injection, the company's investigational opioid antagonist for the emergency treatment of known or suspected opioid overdose. (purduepharma.com)
• The Company's clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. (abeonatherapeutics.com)
• In recent years, Pfizer has made significant investments in the company's gene therapy manufacturing facilities in North Carolina to strengthen its ability to produce both clinical and commercial scale quantities of gene therapies for patients living with rare diseases around the world. (vivet-therapeutics.com)
• The company's patient centric mission is to leverage its proprietary cell and gene therapy platforms and novel business model to rapidly innovate breakthrough immunotherapies for cancers and viral infections with high unmet need. (medtelligence.net)

Efficacy9

• Since the first successful gene therapy for thalassemia major, in 2007, researchers have worked to improve the efficacy and safety of the procedure. (medscape.com)
• This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. (purduepharma.com)
• This hypothesis was confirmed in preclinical studies, conducted under a strategic research collaboration between Merck and Telix announced in 2019, [1] and provided evidence that the combined effect of Merck's investigational DNA-PK inhibitor peposertib with Telix's targeted radiation candidate has potential to significantly improve efficacy and reduce the required radiation dose for tumour reduction and remission, compared to targeted radiation alone. (aap.com.au)
• Neoadjuvant therapy for high-risk resectable melanoma has demonstrated significant efficacy in early clinical trials. (medscape.com)
• These studies also showed that combination intravenous and intrathecal dosing provided incremental efficacy over either delivery route alone and thus may enhance the therapeutic potential of this gene therapy. (abeonatherapeutics.com)
• Several targeted therapies have shown efficacy in patients with advanced gastric cancer (GC) and gastroesophageal junction adenocarcinoma (GEJC), including anti-angiogenic agents and immune checkpoint inhibito. (biomedcentral.com)
• The manuscript, "Anti-BCMA CAR T Cell Therapy bb2121 in Relapsed/Refractory Multiple Myeloma", published in NEJM include key safety and efficacy results from the dose escalation and first expansion cohort, including a minimum of six months follow up on all subjects. (eurekalert.org)
• The Society for Healthcare Epidemiology of America (SHEA) and the Infectious Diseases Society of America (IDSA) recommend a regimen of metronidazole (500 mg PO TID for 10-14 days) as first-line therapy for mild to moderate disease without complications (lower cost and similar efficacy to PO vancomycin in these patients). (medscape.com)
• [ 80 ] The study showed that fidaxomicin therapy had a similar efficacy and resulted in a significantly lower rate of recurrence of CDI not associated with the epidemic North American Pulsed Field type 1 strains. (medscape.com)

Inhibitor7

• WHITEHOUSE STATION, N.J., March 30, 2011 - Merck (NYSE: MRK), known as MSD outside the United States and Canada, announced today that final results from the two pivotal Phase III studies of VICTRELIS (boceprevir), its investigational oral hepatitis C protease inhibitor, will be published in the March 31st edition of The New England Journal of Medicine (NEJM). (natap.org)
• In patients with no BRAF mutation (ie, wild-type BRAF ), current guidelines from the National Comprehensive Cancer Network (NCCN) recommend single-agent immunotherapy with the programmed cell death-1 (PD-1) inhibitor pembrolizumab or nivolumab or combination therapy with nivolumab plus ipilimumab. (medscape.com)
• Under a licensing agreement with Incyte Corporation, Novartis will have responsibility for the future development of Incyte's investigational JAK inhibitor outside the US and for future development of an early-stage cmet inhibitor globally. (hospitalpharmacyeurope.com)
• The lead compound is a Janus kinase (JAK) inhibitor with the investigational name INCB18424. (hospitalpharmacyeurope.com)
• The second compound covered in the licensing agreement, a mesenchymal-epithelial transition factor kinase (cMET) inhibitor with the investigational name INCB28060, is entering Phase I development. (hospitalpharmacyeurope.com)
• This study is looking at how well the drug elimusertib (a type of targeted therapy known as an ATR inhibitor) works when combined with chemotherapy for treating people with advanced cancers, including urothelial, ovarian, gastric, endometrial, triple-negative breast cancer and others. (facingourrisk.org)
• RARITAN, NJ - April 8, 2013 - Janssen Research & Development, LLC (Janssen) announced today that the U.S. Food and Drug Administration (FDA) has granted a third Breakthrough Therapy Designation for the investigational oral Bruton's tyrosine kinase (BTK) inhibitor ibrutinib. (jnj.com)

Treatment39

• The one-time gene therapy was found to give patients the ability to sit, walk, and talk, from data representative of up to five years of follow up post-treatment. (pharmatimes.com)
• In the studies, the addition of VICTRELIS to peginterferon alfa-2b and ribavirin (PR) significantly improved sustained virologic response (SVR)1 - the goal of treatment - for adult patients who failed previous treatment (treatment-failure) and those who were new to treatment (treatment-naïve) for chronic hepatitis C virus (HCV) genotype 1, compared to standard therapy (PR) alone. (natap.org)
• In these studies, nearly half of all patients receiving response-guided therapy with VICTRELIS were eligible for a shorter course of treatment that was 12 to 20 weeks less than the standard 48 weeks of therapy. (natap.org)
• Using this approach enabled many patients in the studies - both those who failed previous treatment and those who were new to treatment - to achieve success with a shorter duration of therapy compared to current therapy," said Fred Poordad, M.D., chief of hepatology and liver transplantation, Cedars-Sinai Medical Center, Los Angeles, and lead author for the HCV SPRINT-2 study in treatment-naïve patients. (natap.org)
• Using response-guided therapy in these studies provided physicians flexibility in the management of their patients' HCV therapy, which enabled them to adapt treatment duration based on individual patient response. (natap.org)
• The HCV RESPOND-2 and HCV SPRINT-2 studies each evaluated two treatment strategies with VICTRELIS added to PEGINTRON (peginterferon alfa-2b) and ribavirin (PR) to assess the ability of VICTRELIS to improve SVR rates and potentially shorten overall treatment duration compared to the use of PR alone for 48 weeks, which is the current standard duration of therapy. (natap.org)
• · Response-guided therapy (RGT), in which total treatment duration was based on certain early response criteria. (natap.org)
• Primary results: Adding VICTRELIS significantly increased SVR compared to control The HCV RESPOND-2 study was conducted at U.S. and international sites, and included 403 adult patients who had failed prior therapy, including patients who relapsed or were non-responders to prior treatment with peginterferon and ribavirin. (natap.org)
• Because the definitive treatment of cutaneous melanoma is surgery, medical management is reserved for adjuvant therapy of patients with advanced melanoma. (medscape.com)
• Gene therapy has been shown to be a simple, efficient, and convenient treatment. (wikidoc.org)
• By providing the allergen or therapeutic protein in the form of plasmid DNA in vivo to modulate allergic immune responses, gene therapy has been shown to be a simple, efficient, and convenient treatment. (wikidoc.org)
• This oral targeted therapy is in Phase III clinical trials for the treatment of myelofibrosis, a life-threatening neoplastic condition with no effective medical treatment that is characterised by varying degrees of bone marrow failure, splenomegaly (enlarged spleen) and debilitating symptoms. (hospitalpharmacyeurope.com)
• Emerging evidence indicates that cMET inhibition may be useful in the treatment of certain cancers, including gastric and kidney cancer, and may help to overcome resistance to some targeted therapies, such as gefitinib in non-small cell lung cancer. (hospitalpharmacyeurope.com)
• This agreement leverages these two promising investigational drugs with Novartis Oncology's global development and commercialisation expertise and our wide range of multi-targeted approaches to cancer treatment. (hospitalpharmacyeurope.com)
• Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders. (wikipedia.org)
• If a safe pharmacological treatment can be developed, one that causes the increased expression of lysosomal sialidase in neurons, a new form of therapy, essentially curing the disease, could be on the horizon. (wikipedia.org)
• Metabolic therapies under investigation for Late-Onset TSD include treatment with the drug OGT 918 (Zavesca). (wikipedia.org)
• In these circumstances, Alnylam will consider providing a requesting physician with access to a specific Alnylam investigational therapy, for the treatment of an individual patient outside of a clinical trial (compassionate use request), or through an Alnylam early access program (EAP) when certain conditions are met. (alnylam.com)
• Early access programs are a means by which investigational therapies are made available, in certain circumstances, to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited. (alnylam.com)
• The frequency and activity of terminally differentiated NKG2a/c low CD16 + natural killer cells correlates with a reduction of replication-competent SIV in lymph node during antiretroviral therapy and time to viral rebound following analytical treatment interruption. (nature.com)
• We hope that this potentially first-in-class BCMA-targeted CAR T-cell therapy may provide a new treatment option for patients living with multiple myeloma. (eurekalert.org)
• The data are highlighted in a poster titled, "CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies," at the Society for Immunotherapy of Cancer's (SITC) 36th Annual Meeting, being held Nov. 10-14, 2021. (beamtx.com)
• However, Astellas recognizes that patients with serious or life-threatening diseases may not qualify for a clinical trial and may seek access to investigational therapy if they have exhausted all available treatment options. (astellas.com)
• Tevogen Bio, a clinical stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for TVGN-489, Allogeneic COVID-19 Specific Cytotoxic T Lymphocytes (CTLs), allowing Tevogen Bio to initiate clinical trial of its investigational COVID-19 treatment. (medtelligence.net)
• NORTH CHICAGO, Ill., May 8, 2014 /PRNewswire/ AbbVie (NYSE ABBV) submitted marketing authorization applications (MAAs) to the European Medicines Agency (EMA) seeking approval for the company' s investigational, all oral, interferon free regimen for the treatment of adult patients with chronic genotype 1 (GT1) hepatitis C virus (HCV) infection. (cigarettesintheusa.com)
• The AbbVie HCV clinical development program is intended to advance scientific knowledge and clinical care by investigating an interferon free, all oral regimen with and without ribavirin with the goal of producing high sustained virologic response rates in as many patients as possible, including those that typically do not respond well to treatment, such as previous non responders to interferon based therapy or patients with advanced liver fibrosis or cirrhosis. (cigarettesintheusa.com)
• ABT 450 is being developed by AbbVie for use in combination with AbbVie' s other investigational medicines for the treatment of hepatitis C. (cigarettesintheusa.com)
• Whereas metronidazole is the recommended first-line treatment for mild to moderate disease without complications, vancomycin is recommended first-line therapy for severe infection or for patients with risk factors for adverse outcomes, such as advanced age, critical illness, overall debility, or inflammatory bowel disease (IBD). (medscape.com)
• Improved tolerance of therapies for advanced HCC may lead to reduction in treatment discontinuation and contribute to better patient outcomes. (ajmc.com)
• Previous work by the group showed that renal VEGF therapy looked like a promising treatment but it had limited effects, perhaps due to VEGF's susceptibility to be degraded. (sciencedaily.com)
• Ibrutinib has been granted Breakthrough Therapy Designation as a monotherapy in the treatment of patients with chronic lymphocytic leukemia or small lymphocytic lymphoma with deletion of the short arm of chromosome 17 (del17p). (jnj.com)
• Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy Designation is intended to expedite the development and review time for a potential new medicine "to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. (jnj.com)
• Appropriate management of therapy and complications is possible only when adequate diagnostic and treatment facilities are readily available. (nih.gov)
• It has been shown to be useful in the therapy of disseminated adenocarcinoma of the stomach or pancreas in proven combinations with other approved chemotherapeutic agents and as palliative treatment when other modalities have failed. (nih.gov)
• Treatment involves controlling triggering factors and drug therapy, most commonly with inhaled beta-2 agonists and inhaled corticosteroids. (msdmanuals.com)
• The treatment of HIV with medicines is called antiretroviral therapy (ART). (medlineplus.gov)
• Title : Intravenous Artesunate for the Treatment of Severe and Complicated Malaria in the United States: Clinical Use under an Investigational New Drug Protocol Personal Author(s) : Twomey, Patrick S.;Smith, Bryan L.;McDermott, Cathy;Marino, Anne;McCarthy, William;Kachur, S. Patrick;Ransom, Janet;Arguin, Paul M. (cdc.gov)
• In the bigger picture, these new treatment options need to be weighed against the possibility of cure of hemophilia through gene therapy: two adeno-associated virus (AAV) vector-based products have been approved for clinical use, and more are in the pipeline. (cdc.gov)
• The Food and Drug Administration approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder. (cdc.gov)

Clinical trial2

• Our Expanded Access Policy (EAP) refers to the use of an investigational cell therapy outside of a clinical trial. (eurekaconnectme.com)
• In rare cases where patients with serious diseases are unable to participate in clinical trials and have exhausted all available options, Eureka may consider providing our investigational therapies outside of a clinical trial. (eurekaconnectme.com)

20221

• Telix's companion investigational diagnostic imaging agent TLX250-CDx ( 89 Zr-DFO-girentuximab) was the subject of a successful global Phase III trial (ZIRCON trial, NCT03849118 ), which completed enrolment and reported positive top-line data during 2022. (aap.com.au)

Merck's3

• Merck and Telix are continuing the initial scientific collaboration to further explore and optimise the combination of TLX250 or other next-generation targeted radiation therapy candidates with Merck's DDRi compounds in preclinical studies. (aap.com.au)
• KENILWORTH, N.J.-( BUSINESS WIRE )-Merck, known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted priority review for Merck's investigational Ebola vaccine (V920), under review for the prevention of disease caused by the Ebola Zaire virus. (merck.com)
• V920, Merck's investigational Ebola Zaire vaccine, was initially engineered by scientists from the Public Health Agency of Canada's National Microbiology Laboratory and subsequently licensed to a subsidiary of NewLink Genetics Corporation. (merck.com)

Drugs2

• Expert Opinion on Investigational Drugs. (wikipedia.org)
• The syndrome may occur at any time during systemic therapy with mitomycin as a single agent or in combination with other cytotoxic drugs, however, most cases occur at doses ≥60 mg of mitomycin. (nih.gov)

Approved therapies that address1

• Unfortunately, there are currently no approved therapies that address the underlying cause, and as such patients with severe AADC deficiency have a high risk of death during childhood. (pharmatimes.com)

Patients29

• Patients receiving long-term transfusion therapy also require iron chelation. (medscape.com)
• We are committed to bringing forward the most impactful new therapies that will improve the lives of our patients," said Ferran Prat , Ph.D., J.D. , senior vice president for Research Administration and Industry Relations at MD Anderson. (biospace.com)
• Through the Gayle and Tom Benson Cancer Center at Ochsner, the Precision Cancer Therapies Program offers the only program in our region dedicated to offering early phase clinical trials for cancer patients," said Leonardo Seoane M.D . Chief Academic Officer and Senior Vice-President Ochsner Health. (biospace.com)
• In both studies, all patients receiving VICTRELIS were treated with a 4-week lead-in of PEGINTRON (1.5 mcg/kg/week) and an investigational dose of ribavirin (600-1,400 mg/day), followed by the addition of VICTRELIS (800 mg three times a day). (natap.org)
• The target population is patients with CAIX-expressing solid tumours that are relapsed or refractory to standard therapies. (aap.com.au)
• consequently, these patients are classified as high risk and should be considered for adjuvant therapy. (medscape.com)
• For patients with a BRAF mutation, the NCCN recommends targeted combination therapy with dabrafenib/trametinib or vemurafenib/cobimetinib. (medscape.com)
• Some patients have multidrug-resistant HIV therapy and have few options left. (mdforlives.com)
• All patients were adults or adolescents (ages 12 and up) who had multi-drug resistant HIV-1 infection, with their current HIV therapy failing to control the infection. (mdforlives.com)
• The study included a placebo-controlled cohort (cohort 1) of 36 patients, who were randomized at a 2:1 ratio to receive either oral lencapavir or placebo, along with their existing ineffective therapy, over 15 days. (mdforlives.com)
• Novartis has gained exclusive rights to therapies for patients with a range of life-threatening blood disorders. (hospitalpharmacyeurope.com)
• However, the patients often generate inhibitory IgG antibodies in their body that act as inhibitors against the therapy. (geneonline.com)
• We look forward to continuing to follow these patients to further understand the durability of response to SB-525 gene therapy and to working with Pfizer to potentially advance a registrational study. (geneonline.com)
• Alnylam is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families, and patient communities through education, empathy, and awareness. (alnylam.com)
• We understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. (alnylam.com)
• SUMMIT, NJ and CAMBRIDGE, MA (May 2, 2019)-- Celgene Corporation (Nasdaq: CELG) and bluebird bio, Inc. (Nasdaq: BLUE) today announced that the New England Journal of Medicine (NEJM ) has published interim results from CRB-401, the ongoing phase 1 study of bb2121, the companies' lead investigational BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy candidate for patients with relapsed and refractory multiple myeloma. (eurekalert.org)
• CAR T-cell therapy is an important area of research for relapsed/refractory multiple myeloma patients where there remains a need for new options. (eurekalert.org)
• We are proud of the significant progress made to date in our gene therapy manufacturing capabilities, and this milestone in our relationship with Vivet underscores the importance of our strategic investments in end-to-end gene therapy research and supply, to support the timely delivery of these important new therapies for patients," said Seng Cheng, Chief Scientific Officer, Rare Disease Research Unit, Pfizer. (vivet-therapeutics.com)
• We believe our approach using multiplex editing of T-cells to produce allogeneic anti-CD5 CAR T investigational therapies may offer enhanced potency and more durable responses for patients with T-cell malignancies. (beamtx.com)
• The general approach for making our therapies available to patients in need is through clinical trials and subsequent registration and commercialization of our products. (astellas.com)
• As appropriate, Astellas commits to establishing expanded and early access routes to investigational therapies for patients that satisfy certain defined conditions in compliance with local regulations. (astellas.com)
• For some therapies, our international import program supports patients seeking access to medicines after approval and before reimbursement in their country. (astellas.com)
• LX1001 is an AAV-based investigational gene therapy designed to deliver the apolipoprotein E2 (APOE2) gene into the CNS of APOE4 homozygous Alzheimer's disease patients to halt or slow disease progression. (lexeotx.com)
• We are excited to see our second program in T1D advancing into the clinic and look forward to bringing transformative, if not curative, therapies to T1D patients who are waiting. (times-online.com)
• For example, patients may need to choose between mastectomy or breast conserving surgery (BCS), whether to have radiotherapy after BCS, what type of radiotherapy (eg, whole breast, partial breast, accelerated partial breast irradiation, boost dose) to have, whether to undergo a lymph node biopsy, and whether to opt for primary endocrine therapy instead of surgery or radiation. (medscape.com)
• Approximately 15-25% of patients respond to conservative therapy, which allows for the reconstitution of normal colonic flora and reduces the risk of relapse. (medscape.com)
• Patients with unresectable or inoperable lesions should be considered for arterially directed embolic therapy, systemic therapy, or radiation. (ajmc.com)
• This third Breakthrough Therapy Designation reflects the potential importance of ibrutinib for patients diagnosed with a 17p deletion chromosomal abnormality in CLL/SLL, and we are committed to working with Pharmacyclics and the FDA to expedite development and review of ibrutinib as quickly as possible. (jnj.com)
• In February 2013 , FDA granted Breakthrough Therapy Designations for ibrutinib as a monotherapy for two B-cell malignancies: in patients with relapsed or refractory Mantle cell lymphoma (MCL) who have received prior therapy, and in patients with Waldenström's macroglobulinemia (WM). (jnj.com)

Allogeneic1

• VX-264 is an investigational cell therapy in which allogeneic human stem cell-derived islets are encapsulated in a channel array device designed to shield the cells from the body's immune system. (times-online.com)

Pharmacological2

• Pharmacological therapies that are safe and fight muscle wasting and fibrosis are still needed. (defeatduchenne.ca)
• investigational pharmacological treatments for Parkinson's disease, William Ondo. (routledge.com)

20192

• For published articles in "Emerging agents and regimens for cancer therapy 2019," see here . (biomedcentral.com)
• In addition, in June 2019, Merck started executing an updated replenishment strategy to increase investigational V920 Ebola vaccine supply, based on ongoing consultations with the U.S. Department of Health and Human Services, WHO and Gavi (the Vaccine Alliance). (merck.com)

Curative1

• Curative therapies for HCC include surgical resection, liver transplantation, and tumor ablation. (ajmc.com)

Treating Duchenne muscular1

• Scientists at Saint Louis University believe that an investigational therapy called SR8278 holds potential for treating Duchenne muscular dystrophy (DMD) after it was shown to increase muscle function and decrease muscle fibrosis in lab mice. (defeatduchenne.ca)

Breakthrough Therapy Designation4

• In November 2017, bb2121 was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration and PRIority Medicines (PRIME) eligibility by the European Medicines Agency based on preliminary clinical data from the phase 1 CRB-401 study. (eurekalert.org)
• The implications of Breakthrough Therapy Designation cannot be determined at this time. (jnj.com)
• Janssen and Pharmacyclics are working with the FDA to determine any potential implications of the Breakthrough Therapy Designation to the ongoing and planned development activities. (jnj.com)
• In July 2016, the FDA granted Breakthrough Therapy Designation to V920. (merck.com)

Therapeutic1

• CAR T therapies have the potential to address a number of T-cell malignancies, but their therapeutic potential is often hindered by technological limitations in their development and application. (beamtx.com)

Gene therapy designed1

• ABO-202 is a one-time AAV gene therapy designed to enable cells to produce the normal PPT1 enzyme, which is critical for proper lysosomal function. (abeonatherapeutics.com)

Trials5

• The Cancer Focus Fund is a unique investment fund that aligns the scientific and clinical expertise of MD Anderson with business experts, public financing entities and private venture capital to propel promising cancer therapies toward pivotal trials," said Ross Barrett , a founder and Managing Partner of Cancer Focus Fund. (biospace.com)
• As part of the partnership, MD Anderson will design and host clinical studies for therapies that receive IND approval, utilizing the extensive expertise of MD Anderson physicians in conducting early stage clinical trials. (biospace.com)
• We are very excited to partner with the Cancer Focus Fund to help develop new therapies to treat cancer and provide these clinical trials closer to our communities. (biospace.com)
• Eureka believes that participation in one of our clinical trials is the best way to access our investigational cell therapies. (eurekaconnectme.com)
• If you are a physician who is interested in learning more about our investigational cell therapies, or participating in our clinical trials, please submit a request to [email protected] . (eurekaconnectme.com)

Autologous1

• As a developer of autologous cellular therapies - treatments that use a patient's own cells and tissues - for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. (alsnewstoday.com)

Designation1

• Vivet's second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020. (vivet-therapeutics.com)

Pipeline2

• LEXEO is advancing a deep and diverse pipeline of AAV-based gene therapy candidates in rare cardiovascular diseases, APOE4 -associated Alzheimer's disease, and CLN2 Batten disease, and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing, and commercialization. (lexeotx.com)
• SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. (synconaltd.com)

20202

• This Journal of Hematology & Oncology collection is the 2020 version of an annual series on emerging agents and regimens for cancer therapy. (biomedcentral.com)
• The companies also announced that they would collaborate on the development of VTX-801, for which an Investigational New Drug (IND) application is planned to be filed with the U.S. Food and Drug Administration (FDA) in 2020. (vivet-therapeutics.com)

Systemic therapy2

• Options for first-line systemic therapy of advanced HCC include sorafenib, lenvatinib, and atezolizumab plus bevacizumab. (ajmc.com)
• New systemic therapy for their cancer (palliative radiation therapy is allowed). (who.int)

Mitomycin1

• Mitomycin for Injection, USP is not recommended as single-agent, primary therapy. (nih.gov)

Treatments1

• The well-timed Third Edition is expanded and topically reorganized to register trends and progress in anti-parkinsonian medications, ablative surgical options, deep brain stimulation, nonmotor symptom treatments, nonmedical strategies, and investigational therapies. (routledge.com)

Serious diseases1

• The company' s mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world' s most complex and serious diseases. (cigarettesintheusa.com)

Regimen2

• The AbbVie investigational regimen consists of the fixed dose combination of ABT 450/ritonavir (150/100mg) co formulated with ombitasvir (ABT 267) 25mg, dosed once daily, and dasabuvir (ABT 333) 250mg with or without ribavirin (weight based), dosed twice daily. (cigarettesintheusa.com)
• If in the investigator*s assessment a change is needed to the subject*s regimen approval (e.g., dose change in androgen deprivation therapy (ADT) or dropping of a combination therapy) approval from a medical monitor is required prior to enrollment. (who.int)

Development7

• There is no guarantee that nalmefene HCl injection, an investigational agent, will successfully complete development or gain FDA approval. (purduepharma.com)
• Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use. (eurekaconnectme.com)
• We continue to be encouraged by the potential of bb2121 as a first-in-class BCMA-targeted CAR T-cell therapy," said Alise Reicin, M.D., President, Global Clinical Development for Celgene. (eurekalert.org)
• SBT101 is the first AAV-based gene therapy in development specifically designed for people living with AMN, an adult-onset degenerative spinal cord disease caused by mutations in the ABCD1 gene. (synconaltd.com)
• SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy (AMN). (synconaltd.com)
• Merck has worked with government partners and the global health community to accelerate development of our investigational V920 Ebola vaccine. (merck.com)
• point out, the rapid development of new therapies will greatly impact newborn screening programs as the number of candidate diseases for screening will very likely increase at a pace not sustainable by the current system. (cdc.gov)

Tumor3

• Current targeted therapies can slow tumor growth (eg, BRAF inhibition) or release the brakes on the immune response, resulting in tumor lysis (eg, PD-1 inhibition). (medscape.com)
• Compounds in this class are envisioned to become effective cancer therapies through their ability to block molecular signals leading to tumor cell angiogenesis, proliferation, survival, invasion and metastasis. (hospitalpharmacyeurope.com)
• Individualized neoantigen therapies are designed to prime the immune system so that a patient can generate an antitumor response specific to their tumor mutation signature. (malvern-online.com)

Cell8

• If you are a patient who is interested in accessing our investigational cell therapies, please speak with your physician. (eurekaconnectme.com)
• Unlike HIV infection, which progresses to AIDS absent suppressive anti-retroviral therapy, nonpathogenic infections in natural hosts, such African green monkeys, are characterized by a lack of gut microbial translocation and robust secondary lymphoid natural killer cell responses resulting in an absence of chronic inflammation and limited SIV dissemination in lymph node B-cell follicles. (nature.com)
• furthermore, in vivo IFNα-induced NK cell cytotoxicity correlates with reductions in HIV-DNA during antiretroviral therapy (ART) 16 . (nature.com)
• Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes (T1D). (times-online.com)
• VX-264 uses the same stem cell-derived pancreatic islet cells as our VX-880 program where we've already demonstrated proof-of-concept, with the addition of a proprietary immunoprotective device that allows us to eliminate the need for immunosuppression," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. (times-online.com)
• Vertex is pursuing multiple investigational approaches using stem cell-derived islets with the aim of replacing the insulin-producing islet cells that are destroyed in people with T1D. (times-online.com)
• Vertex's first clinical investigational program in T1D, VX-880, is a stem cell-derived, fully differentiated, insulin-producing islet cell replacement therapy used in combination with immunosuppression. (times-online.com)
• Currently, the FDA expects to receive 200 investigational new drug applications annually and anticipates soon approving 10-20 cell and gene therapies each year . (cdc.gov)

Accelerate2

• The goal is to accelerate the progress of these promising therapies through Investigational New Drug (IND) approval and Phase I and Phase Ib/II studies. (biospace.com)
• New investigational supply will be based on a combination of leveraging material from ongoing production activities at the planned commercial manufacturing site in Germany and new production at a clinical manufacturing site in the U.S. While the company continues to explore opportunities to accelerate production, our timing estimates are based on the need to meet manufacturing and quality-control requirements. (merck.com)

Pivotal1

• Evaluating response-guided therapy with VICTRELIS was an important component of these pivotal studies. (natap.org)

Clinically1

• [ 21 ] ​ Targeted therapy is preferred if clinically needed for early response. (medscape.com)

Infection1

• Pharmacologic therapy should be tailored according to the severity of C difficile infection (CDI). (medscape.com)

Antiretroviral2

• Here we report, using the pathogenic model of antiretroviral therapy-treated, SIV-infected rhesus macaques that sequential interleukin-21 and interferon alpha therapy generate terminally differentiated blood natural killer cells (NKG2a/c low CD16 + ) with potent human leukocyte antigen-E-restricted activity in response to SIV envelope peptides. (nature.com)
• What is antiretroviral therapy (ART)? (medlineplus.gov)

Approach2

• Transplantation-related issues such as graft versus host disease, graft failure, chronic immunosuppressive therapy, and transplantation-related mortality should be carefully considered prior to proceeding with this approach. (medscape.com)
• We also believe our unique approach has the potential to reward our investors and thereby provide incentives for further investments in innovative cancer therapies. (biospace.com)

20182

• Since May 2018, Merck has donated and shipped more than 245,000 1.0mL investigational V920 Ebola vaccine doses to the World Health Organization (WHO) in response to requests by the WHO. (merck.com)
• The availability of innovative therapies for SMA, including gene therapy, enabled it to be added to the RUSP in 2018. (cdc.gov)

Dose1

• The data showed that the therapy was generally well-tolerated and demonstrated a dose-dependent increase in fVIII activity. (geneonline.com)

Severe1

• As noted above, for severe cases, vancomycin (125 mg PO QID for 10 days) is the recommended first-line therapy. (medscape.com)

Potential2

• Without appropriate support, we know that some therapies with great potential may be delayed, may not be developed properly in the clinical setting or may never make it into clinical studies. (biospace.com)
• Lenacapavir is a first-in-class potential HIV therapy that interferes with both early and late capsid functions, thus attacking the virus at two points in its life cycle. (mdforlives.com)

Safety2

• This study is designed to test the safety of 4D-710, an investigational gene therapy drug for adults with cystic fibrosis (CF) who are unable to use CFTR modulators. (nationaljewish.org)
• This study is comparing safety and effectiveness of a new drug called giredestrant compared to standard hormone therapy in people with early-stage, ER-positive breast cancer. (facingourrisk.org)

Study1

• Phase I multicenter, nonrandomized, investigational device study. (nih.gov)

Continues1

• Access to the investigational therapy will generally continue as long as the patient continues to receive benefit as determined by the treating physician and will end should the therapy become commercially available for the relevant indication in the patient's country of residence. (alnylam.com)

Commercially available1

• Astellas considers the feasibility of product donation and voluntary licensing programs, through external non-profit/non-governmental organizations, for certain therapies as a route to make our therapies accessible in countries where they are not commercially available. (astellas.com)

Cancer1

• Also at the session, Jennifer Tseng, MD, medical director of breast surgery at City of Hope Orange County Cancer Center, Irvine, California discussed de-escalation of locoregional therapy. (medscape.com)

Program3

• Additionally, our international pharmacy program can facilitate access to certain therapies by importing products approved in major countries, in countries where it is not yet approved. (astellas.com)
• This marks the first clinical data from a gene therapy program targeting APOE4 -associated Alzheimer's disease presented at a scientific meeting. (lexeotx.com)
• The VX-264 program does not require the use of immunosuppression, which may broaden the population of people with T1D that this investigational therapy could reach. (times-online.com)