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CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. In 2018, the company also received approval for Orilissa (elagolix), which it licenses to AbbVie, for the treatment of endometriosis. billion in revenue by 2024.
Identifying branded drugs with a low likelihood of generic entry has become a crucial strategy for companies looking to expand their product portfolio through in-licensing. In this comprehensive guide, we’ll explore the intricacies of identifying such drugs and leveraging them for successful in-licensing opportunities.
A licensing deal with Jiangsu Hengrui Pharmaceuticals puts Merck in a competitive race to develop a medicine that targets a genetic risk factor called lipoprotein(a).
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).
Jnana said in a statement that the funding will allow it to take JNT-517 through proof-of-concept testing in PKU, a genetic disorder caused by an inability to break down the amino acid phenylalanine in the diet that can lead to serious neurological and neuropsychological symptoms if untreated.
These efforts aim to fast-track commercialization and out-licensing agreements in one of the world’s most promising pharmaceutical markets. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.
MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.
Related: FDA Approves New Use for Ozempic to Reduce the Risks of Kidney Disease Maze stands out in the biotech space with its Compass platform, which analyzes genetic data to identify disease-linked variants. For complex diseases like chronic kidney disease (CKD), where genetics play a significant role, such a platform holds promise.
Innova Medical Group, the world’s largest COVID-19 test provider, has secured the licensing rights for commercializing a new COVID-19 testing technology developed at the University of Birmingham. Innova Medical Group, which secured the licensing rights for the test, is the world’s largest provider of lateral flow tests.
ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.
The British investment trust Syncona Limited launched the new ophthalmic gene therapy company by combining Applied Genetic Technologies Corporation’s (AGTC’s) late-stage X-linked retinitis pigmentosa (XLRP) programme with two proprietary preclinical programmes. Beacon Therapeutics is the third gene therapy company Syncona has launched.
Opus Genetics Announces Agreement with Massachusetts Eye and Ear and Harvard Medical School to License Third Program for Inherited Retinal Disease Opus Genetics Announces Agreement with Massachusetts Eye and Ear and Harvard Medical School to License Third Program for Inherited … Continue reading →
Brooklyn ImmunoTherapeutics Pays $1 Million Towards Acquisition of License for mRNA Technology Platform to Develop Genetically Edited Cells for Multiple Cancers and Blood Disorders Brooklyn ImmunoTherapeutics Pays $1 Million Towards Acquisition of License for mRNA Technology Platform to Develop Genetically … Continue reading →
Alnylam’s Onpattro was its first licensed and approved RNAi therapy for ATTR polyneuropathy. Results of the study show that the RNAi therapeutic significantly improved the signs and symptoms of polyneuropathy and 50 percent of patients achieved a cease or reversal of their disease manifestations.
BEIJING — Hong Kong on Tuesday revoked a visa it granted to a Chinese scientist who set off an ethical debate five years ago with claims that he made the world’s first genetically edited babies, pulling it hours after he announced his research plans in the financial hub. Read the rest…
Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. sickle cell disease community and the millions around the globe who suffer from this genetic illness. In June 2023, the U.S.
Under the multi-year strategic partnership, Prevail will detect and advance capsids, which are clinically translatable, along with its cargo to develop the transformative genetic medicines by using Capsida’s novel adeno-associated virus (AAV) engineering platform.
Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. High Lp(a) levels can double or even triple the risk of a heart attack.
Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D).
The US Food and Drug Administration (FDA) has granted priority review to Roche’s Biologics License Application (BLA) of Lunsumio (mosunetuzumab) to treat adults with relapsed or refractory (R/R) follicular lymphoma (FL). These patients should have received a minimum of two prior systemic therapies.
ImmunoForge chief technology officer Dr Jim Balance pioneered the clinical development of peptide drugs genetically fused to ELPs. ImmunoForge will expedite the development of new drugs based on the elastin-like polypeptide (ELP) platform technology originally developed by Duke’s Professor Ashutosh Chilkoti.
MSD has signed two new oncology-based deals with Seattle Genetics which together could be worth up to around $4.4 For this, MSD will put up $600 million up front and a $1 billion equity investment for five million of Seattle Genetics’ shares, while the latter will also have the option to scoop up an additional $2.6 Matt Fellows.
Lacerta Therapeutics has entered a new licensing and research partnership agreement with Eli Lilly and Company subsidiary, Prevail Therapeutics, to discover and develop adeno-associated virus (AAV) capsids for treating central nervous system (CNS) diseases. .
ERS Genomics and Japan SLC Sign CRISPR/Cas9 License Agreement ERS Genomics and Japan SLC Sign CRISPR/Cas9 License Agreement Nobel prize winning CRISPR technology to be applied in development of genetically engineered animal models DUBLIN & SHIZUOKA, Japan–(BUSINESS WIRE)–ERS Genomics Limited, … Continue reading →
ERS Genomics and Setsuro Tech Sign CRISPR/Cas9 License Agreement ERS Genomics and Setsuro Tech Sign CRISPR/Cas9 License Agreement Nobel prize winning CRISPR technology to be applied in development of genetically engineered cell and animal models DUBLIN & TOKUSHIMA, Japan–(BUSINESS WIRE)–ERS … Continue reading →
AZ has previously licensed rights to several other Ionis antisense drugs – including candidates for cholesterol-lowering, non-alcoholic steatohepatitis (NASH), chronic kidney disease, and cancer – but this is the first time that it has gone after a candidate already in late-stage development.
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
Nuvaxovid is a protein-based vaccine created from the genetic sequence of the initial SARS-CoV-2 virus strain. SK bioscience is manufacturing Nuvaxovid’s drug substance, and drug product for domestic use after the companies signed a licensing agreement in Korea.
AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein. Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis.
In 2016, it was licensed by the company to Amgen. The siRNA therapy is created to reduce lipoprotein(a) [Lp(a)] levels, which is a genetically decided, independent cardiovascular disease risk factor. The rights to development, regulatory and sales milestone payments of $400m due from Amgen will be retained by Arrowhead.
The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.
It is also licensed by Spain’s Centro de Investigación Biomédica en Red de Enfermedades Raras, the Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz and the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas. The therapy has also received orphan drug and fast-track designations.
In July, the US Food and Drug Administration (FDA) approved Horizon’s supplemental Biologics License Application for Krystexxa (pegloticase) plus methotrexate, to aid uncontrolled gout patients attain a complete response to treatment.
According to the US biopharma company, genetic analyses of publicly available sequences for the new variants of SARS-CoV-2 suggest the mutations in the viral spike protein that have made them more transmissible shouldn’t affect the way Veklury (remdesivir) works. “We
Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.
The Paleo certification program is based on the organization’s principle of encouraging the avoidance of dairy products, genetically modified organisms (GMO’s), grains, highly processed foods and legumes, among other foods. Licensing is available through The Valen Group, the organization’s exclusive licensing partner.
– Data Featured in Late-Breaking Proffered Paper Oral Presentation – – Biologics License Application Submission Planned to Support Accelerated Approval Pathway with the FDA – BOTHELL, Wash. & & COPENHAGEN, Denmark–(BUSINESS WIRE)–Seattle Genetics, Inc.
KaliVir, Astellas Pharma forms a licensing deal for VET2-L2 oncolytic virus. KaliVir Immunotherapeutics and Astellas Pharma entered into a worldwide exclusive licensing agreement for the development, research, and commercialization of VET2-L2 to widen the horizon of therapeutic approaches available in the Immuno-Oncology market.
Once selected, Quris can in-license them, progress them through to phase 1 trials and, those that succeed, can be licensed to pharma companies. Once the initial drug candidate is licensed to a pharma company, the profits are then shared with the academic institution or biotech from which Quris originally licensed the drug.
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