daniel-gordon/biopharma-dive huggingface dataset:
For 6 months after administration of ROCTAVIAN men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception and men must not donate semen
Half of the companies that have completed an IPO in 2023 had a drug in midtolate stage clinical trials. In 2020 and 2021 when biotech IPOs boomed many companies were going public before they had even begun human testing of their drugs
Forwardlooking statements are based on managements current expectations and aresubject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forwardlooking statements
What were finding with these machine learning tools is we can survey a much more broad spread of potential combinations of proteins and therefore find the optimal answers to specific biological challenges
Additionally people with the most severe form of hemophilia A FVIII levels ULN
AntiTL1As are a promising class of therapies and we believe that TEV574 could emerge as a bestinclass option for people living with serious gastrointestinal diseases said Sanofi CEO Paul Hudson in a statement
The link to the live webc ast will also be available on the Investors  News section of the Ascendis Pharma website at httpsinvestorsascendispharmacom
VectorY plans to use the proceeds to develop its vectorized antibody platform for neurodegenerative diseases. In particular the biotech will push its most advanced program dubbed VTx002 and for amyotrophic lateral sclerosis or ALS into clinical testing
Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability
Shares in Ardelyx rose by around 15 in Wednesday morning trading on news of the approval
QA  Emerging biotech Novoʼs Uli Stilz on biotech innovation and the future of diabetes drugs The Danish drugmakerʼs innovation hub in Boston has teamed up with the Broad Institute Mass General Brigham Harvard and Yale expanding its research into cardiometabolic diseases
AMONDYS 45 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental
It'll also continue a collaboration with gene editing biotech Beam Therapeutics to develop treatments for certain immune system driven diseases
On a nonGAAP basis the net loss for the three months ended June 30 2021 was 1212 million or 152 per basic and diluted share compared to a net loss of 1179 million or 151 per basic and diluted share for the same period of 2020
The latest study evaluated pamrevlumab in Duchenne patients that can still walk. FibroGen said that the drug missed all of its main and secondary goals failing to provide a meaningful improvement over placebo in motor function and other tests
Even in a year when more than 100 biotech companies have laid off staff, Sages action stands out
The deal brings  an entirely new capability to Ajinomoto said the company which known for its production of frozen foods and seasonings such as MSG as well as its work with semiconductors and amino acids. Its backers include Perceptive Advisors RA Capital Management Drive Capital and Surveyor Capital a part of Citadel
VOXZOGO a Ctype natriuretic peptide CNP analog acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth
RayzeBio previously raised 418 million to fund its development of radiopharmaceuticals which though tricky to make can more precisely deliver radiation into tumors
Its initial plan is to develop an experimental drug to treat the inherited disorder alpha1 antitrypsin deficiency or AATD
These enzymes are also involved in asthma and other autoimmune conditions which make those diseases possible targets for TRV 509 in the future said Jennifer Dovey Trivenis chief scientific officer. Its the size and way it can interact with a protease that makes antibodies the perfect modality to target these types of targets in a very selective and potent way Dovey said
The Menlo Park California drug developer was backed by Longitude Capital Enavate Sciences Samsara BioCapital NuevaBio and Eli Lillys venture arm
Based on the meeting with OTAT Sarepta anticipates initiating Study 301 as submitted to the Division in September of this year
Abecma the multiple myeloma treatment Bluebird developed and licensed to Bristol Myers Squibb was supposed to provide a revenue cushion for 2Seventy and help fund its other drug research. But Bristol Myers has had trouble manufacturing supplies and is dealing with strong competition from a rival therapy sold by Johnson  Johnson
Genesis Series B backers also included Fidelity Management BlackRock and Nvidias venture capital arm along with previous investors T Rowe Price Rock Springs Capital Radical Ventures and Menlo Ventures
Shares in Ventyx fell by 80 in Tuesday morning trading erasing hundreds of millions of dollars in market value
Investor Contacts Media Contact Tim Lee Melinda Baker Ascendis Pharma Ascendis Pharma 1 650 374 6343 1 650 709 8875 tleascendispharmacom mediaascendispharmacom irascendispharmacom Patti Bank ICR Westwicke 1 415 513 1284 pattibankwestwickecom
While Ocaliva first gained approval for PBC the drugs potential in the liver disease NASH drove investor enthusiasm in Intercept
The events were asymptomatic and have resolved with magnesium supplementation
Greenstreet added the company still believes it can meet its goal of profitability by 2025 but will need vutrisiran to succeed in HELIOSB
In 2018 Novartis offloaded the other buparlisib  to Adlai Nortye then a privately held Chinabased startup. Novartis did so despite having studied buparlisib in dozens of trials and over 4200 patients
Analyzing the signals in some cases using machine learning may identify cancers early and enable patients to receive treatment while their chances of longterm survival are high. Flagship Pioneering the venture capital fund that created Moderna launched Harbinger in 2020 believing that it has a better approach to multicancer early detection
Kinnate has been working to develop a combination of the two drugs to treat individuals with BRAFaltered solid tumors or those with NRAS mutant melanoma with initial data published in published in April
The other Qalsody is for a small segment of the ALS population whose disease is caused by mutations in a gene called SOD1
The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year
The group of advisers agreed voting 171 that there hadnt been enough evidence to demonstrate NurOwn is effective in treating mild to moderate ALS
New startup Arialys takes aim at the immunesystem  hoping to create neuropsychiatry drugs Backed by Johnson Johnsonʼs venture arm among others Arialys plans to take a medicine it acquired from Astellas Pharma into clinical testing next year
VOXZOGO is the only approved treatment for children with achondroplasia
Using different analyses the company claimed signs of efficacy for izokibep and a consistent safety profile noting most of the discontinuations were unrelated to side effects
The company claims its technology allows for a unique approach to discovering smallmolecule molecular glues that work against hardtodrug disease targets
Artificial intelligence tools have drawn the interest of many drugmakers hoping to use it to unearth new therapeutic targets
Existing liquid biopsy technologies have impacted millions of patients with cancer over the last decade and the precision medicine application space for Precede transcends that of even the most successful of these liquid biopsy technologies creating a significant opportunity for us to impact the practice of medicine. Precede has yet to publish papers to support its claims but will share scientific data later this month
Forward looking statements do not reflect the potential impact of any future licensing collaborations acquisitions mergers dispositions joint ventures or investments that Ascendis may enter into or make
A drug from Pfizer called tafamidis and marketed as Vyndaqel and Vyndamax is currently available for ATTR amyloidosis patients with heart symptoms
AstraZeneca seeking new drug targets taps a genomics biotech and its AI technology. The collaboration will use Verge Genomicsʼ platform which has developed a novel candidate for ALS using data from human tissue
No conclusions can or should be drawn regarding its safety or eectiveness in treating cardiomyopathy in this population
Risks regarding our business are described in detail in our Securities and Exchange Commission SEC filings including in our Annual Report on Form 10K for the fullyear ended December 31 2022  and our Quarterly Report on Form 10Q for the quarter ended June 30 2023  both of which are available on the SECs website at wwwsecgov
The FDA cleared Intellia Therapeutics to begin a latestage trial of a gene editing treatment for the rare disorder transthyretin amyloidosis
Other adverse reactions that occurred at a frequency greater than 5 of VYONDYS 53treated patients and at a greater frequency than placebo were administration site pain back pain pain diarrhea dizziness ligament sprain contusion influenza oropharyngeal pain rhinitis skin abrasion ear infection seasonal allergy tachycardia catheter site related reaction constipation and fracture
A lifelong illness sickle cell can cause strokes organ damage and episodes of intense pain known as vasoocclusive crises. Just a few treatments for the disease are available and the only known cure is a bone marrow transplant
That approach makes Star one of several hub-and-spoke biotechs which typically own a network of drugmaking subsidiaries or experimental medicines that can be hived off and sold individually
Markers of kidney function have generally been normal and not shown any consistent relationship to the hypomagnesemia
Based on Nobel Prizewinning science RNAi therapeutics represent a powerful clinically validated approach yielding transformative medicines
FibroGenʼs Duchenne drug fails second trial in latest study setback The biotech has seen its share value collapse in recent months as the medicine pamrevlumab missed the mark in three latestage trials
Important Safety Information for VYONDYS 53 Hypersensitivity reactions including rash pyrexia pruritus urticaria dermatitis and skin exfoliation have occurred in VYONDYS 53treated patients some requiring treatment
Eight biotech companies priced IPOs from July through the end of September raising 11 billion in total. That compares to four in the second quarter which brought in about 580 million
And in May Moderna released early positive data on one of its rare disease therapies
The company will also prioritize the experimental brain drugs SAGE718 and SAGE324 at the expense of certain less advanced candidates
Our Clinical Co-Development program creates bespoke investment and operational solutions that enable resource constrained pharma companies to pursue additional clinical projects and biotech companies to avoid nearterm equity dilution
Beam will generate a focused clinical dataset for the most advanced of those a leukemia treatment dubbed BEAM201
RayzeBio a San Diego-based startup developing radiopharmaceutical drugs for cancer is the latest biotechnology company to take a shot at going public in a market that is largely been unwelcoming to new stock offerings
If a hypersensitivity reaction occurs institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy
MapLight said the fresh funds will help push the companys most advanced program into midstage clinical trials next year where itll be tested as a potential treatment for schizophrenia and Alzheimers disease psychosis
That treatment called ITCA 650 helped Intarcia raise more than 1 billion in private financing
Additional information will be made available in other filings that we make from time to time with the SEC
But the magnitude of efficacy reported was less than what Ventyx had targeted leading the company to terminate that trial as well as another in psoriatic arthritis
Secondary endpoints include patient assessments by clinicians and caregivers using the Clinical Global Impressions Scale  Severity CGIS the Rett Syndrome Behavior Questionnaire RSBQ and other clinical assessment scales
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients
Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made
Doctors suspect an additional two patients have suffered from retinal vasculitis after injections with the drug but the cases havent been confirmed
But the drugs approval came with questions Syfovre fell short of its main goal in one of the two key studies prompting Apellis to file more data with the FDA before approval
Ingrezza should help address an unmet need in the treatment of Huntingtonsassociated chorea according to Charles Duncan an analyst who covers Neurocrine for the investment firm Cantor Fitzgerald
Agency staff were also unconvinced by retrospective evidence BrainStorm claims show NurOwn helped certain patients noting positive conclusions
Initial public offerings previously a surefire way for growing drug startups to bring in new cash have been harder to pull off this year, while a yeartodate slump in biotech stocks has made it more difficult for public companies to rely on secondary stock offerings
For the six months ended June 30 2021 the Company recorded net product revenues of 2668 million compared to net product revenues of 2118 million for the same period of 2020 an increase of 550 million
The round was led by Canaan and Droia Ventures and also involved existing investors Deerfield Management and EcoR1
Results from the first adult patient dosed in cohort one low dose with TSHA102 in the REVEAL Phase 12 trial
Following treatment we have observed improvements in breathing patterns vocalization and motor skills
We intend to move as fast as science will permit to confirm the benefits of SRP9001 and if successful to rapidly advance this therapy to waiting Duchenne patients around the world Second Quarter 2021 and Recent Corporate Developments Sarepta announced that it concluded its endofphase2 meeting with OTAT In July of 2021 Sarepta completed a successful endofphase2 meeting with the United States Food and Drug Administrations Office of Tissues and Advanced Therapies OTAT regarding the Companys proposed pivotal trial Study SRP9001301 EMBARK and the commercially representative material to be used in that trial
Aiolos approach is similar to that of Upstream Bio  which launched in June 2022 and raised new funds earlier this year. Upstream is also targeting TSLP and announced positive Phase 1 results Tuesday. Upstream said the study shows high potency for its drug UPB101  and the company will explore dosing every 12 weeks and every 24 weeks in a Phase 2 trial for severe asthma
BUSINESS WIREMar
Your healthcare provider will adjust the dose based on changes in weight following regular checkups
A trial in Duchenne patients who can no longer walk failed in early June followed by a setback in the IPF study weeks later. The company then stopped a second IPF trial 2021
In Part 1 results from the treatment and placebo groups are compared through 48 weeks following treatment
If approved we are planning our first EU launch in Germany in early 2024
The unraveling of the agreement which was announced Aug 1  deprives ProQR of immediate cash as well as possible future the market Théa had agreed to an initial payment of 125 million euros or about 132 million plus as much as 135 million euros if certain developmental regulatory and commercial goals were met
Genetic medicines from Bluebird Bio and Vertex Pharmaceuticals have shown that  boosting levels of fetal hemoglobin can in turn lower the rate of vaso occlusive crises. Yet Fulcrums drug while having the convenience of a pill boosts fetal hemoglobin in a different way by blocking a component of an assembly of proteins collectively known as PRC2
RNA editing is a much more accessible approach Elverum said if you think through what a patient wants in terms of the risks they want to take or even what a payer wants in terms of how medicines are paid for The company has not disclosed what other diseases it plans to work on outside of AATD but said its medicines would target both AIRNA has a uniquely powerful technology to maximize the therapeutic properties of RNA editing medicines and achieve the vision of restoring a patients health Novak said in a statement
Apellis emphasized that that the total occurrence of the condition is rare about 0.01% per injection based on more than 100000 vials of the medicine that have been distributed either in trials or commercial sales
Patient Support Accessing VOXZOGO To reach a BioMarin RareConnectionsCase Manager please call toll free 1833VOXZOGO 18338699646 or email VOXZOGOSupportbiomarinrareconnectionscom
Even so the lack of treatment options for geographic atrophy meant that many patients were desperate for a new medicine
Huntingtons disease has proven nearly as difficult as Alzheimers to treat with approved medicines so far only able to reduce involuntary movements. Ionis and Roche are among several although their drug tominersen has had setbacks  too
About Duchenne Muscular Dystrophy Duchenne muscular dystrophy DMD is a rare fatal neuromuscular genetic disease that occurs in approximately one in every 35005000 newborn males worldwide
Cases have resolved with magnesium supplementation and an analysis of all available data indicate that the hypomagnesemia is monitorable and manageable
The firm is still bullish about platform companies  which focus on applying a fundamental technology across a variety of drugs and disease targets
FibroGen has been struggling to regain its footing ever since the Food and Drug Administration in 2021 rejected its anemia pill roxadustat which is approved in Europe and some other countries
Exon skipping was measured by digital drop polymerase chain reaction ddPCR
16 2023 Sarepta Therapeutics Inc NASDAQSRPT the leader in precision genetic medicine for rare diseases today announced that at its late cycle meeting for the SRP9001 delandistrogene moxeparvovec biologics license application BLA the US Food and Drug Administrations Office of Therapeutics OTP has determined that an advisory committee meeting will be held for SRP9001 in advance of the May 29 2023 regulatory action date
Reporter: Why is your company in a good position for novel drug development. CEO: We have always been a consumer company but the goal has always been how are we empowering individuals and how are we changing the trajectory of healthcare, and change it from empowering individuals to get access to their own genetic information as well as changing by helping leverage crowdsourcing data so that we can cure disease
Per deal terms Roche is paying 60 million upfront for rights to the undisclosed drugs and will assume responsibility for developing them once they are ready for human trials. Ionis could receive further payments if clinical regulatory and commercial milestones for the drugs are met
About SRP9001 rAAVrh74MHCK7microdystrophin SRP9001 is an investigational gene transfer therapy intended to deliver the microdystrophinencoding gene to muscle tissue for the targeted production of the microdystrophin protein
Several JAK inhibitors are approved but have safety concerns that TYK2 blockers might sidestep
The FDA approved Xphozah to reduce levels of phosphorus in the blood in adults with chronic kidney disease who are on dialysis and dont respond well to drugs designed to block phosphorus absorption
About 28 million people in the US suffer from schizophrenia and almost a third dont respond to currently available therapies per Karuna
When Rosana Kapeller was working at Nimbus Therapeutics in the early 2010s the technology to study the socalled dark genome didn't exist. Fourteen years later Kapeller is running a biotechnology startup well on its way to shedding light on how those lessexplored stretches of DNA could yield new drugs
Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population
All 41 participants have completed their Part 1 48week assessment and have entered the Part 2 crossover phase
The primary endpoint is safety and secondary and exploratory endpoints include exonskipping dystrophin expression and tissue concentration
Jeffries lowered its estimate for peak sales of the drug to about 700 million from 135 billion largely based on the new data
Sangamo to cut jobs again with planned business overhaul. The gene therapy developer will move headquarters and reduce its workforce by 40  while seeking partners for its Fabry and cell therapy research
MapLight says it has two more medicines in human testing. One is being evaluated as a potential treatment for dyskinesia or involuntary body movements. The other has progressed into Phase 2 trials of patients with autism spectrum disorder
Now the biochemist directs earlystage research collaborations including a deal announced in September with Evotec to launch a drug accelerator as well as a partnership with the Broad Institute on three programs targeting diabetes and other cardiometabolic diseases
At every time point measured the cohort of SRP9001 treated participants outperformed the placebo group and at 48 weeks participants in the treatment group demonstrated a statistically significant increase of 17 points in NSAA total score compared to baseline P0009 while participants in the placebo group saw an increase of 09 points on the NSAA total score compared to baseline which was not statistically significant n21 P01411
In July a safety committee advising the American Society of Retinal Specialists warned doctors about cases of occlusive retinal vasculitis  triggering a selloff of Apellis shares
Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein
Bristol Myers pays 100M for a different kind of ADC A deal with Orum Therapeutics gives the large pharma access to an experimental blood and bone cancer drug thatʼs ready to enter clinical testing
ProQR Therapeutics plan to divest a once-promising eye medicine program faltered after the buyer Laboratoires Théa failed to reach employment agreements with key ProQR personnel.
The FDA also homed in on a small number of major depressive patients who reported having suicidal thoughts and behavior after either receiving Zurzuvae or going off the drug
Recent Corporate Highlights 150 million private placement financing strengthens balance sheet and together with existing cash and cash equivalents extends cash runway into the third quarter of 2025
While collaboration revenue is recurring as the Companys ordinary activities do not include contracting with third parties to provide them with research and development services collaboration revenue is treated as a nonGAAP adjustment item
Histogen intends to close down after an investment bank found no viable options to provide the regenerative medicine company with the money needed to stay operational
Recently the FDA for the second time rejected a wouldbe NASH treatment from the fields former leader Intercept Pharmaceuticals spurring that company to lay off staff and abandon its research
While current treatments act on moodregulating chemicals such as dopamine, KarXT focuses on a neurotransmitter called acetylcholine that affects brain function
Originally developed by the biotech Aimune Palforzia works by gradually desensitizing people who are allergic to peanuts
Liver drug developer Intercept Pharmaceuticals has agreed to be acquired by Italian company Alfasigma for about 800 million the companies announced Tuesday  ending one of biotechnologys most noteworthy stories of investor exuberance and clinical disappointment in the last decade
Cod-evelopment investments are typically around 30 million according to the company. Abingworth also backs earlystage companies though it wont with the latest funds