Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion.

Some say volatility, rather than debt, is the best way to think about risk as an investor, but Warren Buffett famously...

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 24, 2020 -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation.

ProQR Therapeutics N.V. (NASDAQ:PRQR) shareholders might be concerned after seeing the share price drop 11% in the...

Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in.

Vertex's (VRTX) Kalydeco gains EU nod for use in infants with cystic fibrosis aged from six to less than 12 months with at least one of specified nine mutations in the CFTR gene.

LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 30, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced that it received Rare Pediatric Disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for QR-421a for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene.

Editas (EDIT) progresses well on developing its lead candidate EDIT-101 to treat LCA10, a rare genetic illness that causes blindness. Excessive reliance on partners for revenues remains a concern.

LEIDEN, Netherlands and CAMBRIDGE, Mass., Nov. 21, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced that it received Orphan Drug designation (ODD) from the Food and Drug Administration (FDA) for QR-1123. QR-1123 is a first-in-class investigational antisense oligonucleotide designed to address the underlying cause of vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene. ODD provides a special status for investigational drugs being developed for rare diseases.

Risk and reward are two of the most commonly used words in investing. The two are symbiotic and complement each other perfectly. Where there is more risk, the reward is likely to be more abundant. Of course, the equation works the other way too: less risk, probably less reward.The thesis is simple: a company such as Google is likely a pretty safe place for an investment. But with a market cap of $1 trillion, how much value can the company add to its share price? On the other hand, investing in a fledgling biotech with life-changing treatments in the pipeline could potentially see an investment yield extraordinary returns. Let’s not forget, though, this sort of investment also carries a significant amount of risk with it.With this in mind, TipRanks’ Stock Screener pointed us in the direction of three Healthcare stocks the experts on the Street think could soar in the year ahead. And by soar, we mean at least double their current value. The risk here as an investor is higher, but as we know by now, so are the possible rewards. As an added bonus, all three currently have a Strong Buy consensus rating from the Street. Let’s take a look at the data.ProQR Therapeutics (PRQR)ProQR’s back story is unusual. The company was founded after CEO Daniel de Boer’s newborn son was diagnosed with cystic fibrosis. De Boer, with the help of several co-founders, then formed ProQR – a biotech with a focus on providing new medicines for rare diseases. There are approximately 7,000 rare diseases in the world today, and only 400 currently have treatments, a situation ProQR hopes to change with the use of RNA therapies.The company has a diverse portfolio of therapies all in different stages of development. Among these are Sepofarsen, a treatment for Leber congenital amaurosis (LCA-10), the most common genetic cause of childhood blindness. Sepofarsen works by repairing the genetic defect in the RNA, with it standing to fill a gap as there aren’t any approved treatments available. The FDA and EMA have granted the drug orphan designation, and top-line data from the on-going Phase 2/3 Illuminate trial is expected during the first half of 2021. The company is also developing QR-421a, a treatment for Usher syndrome, a rare genetic disease that is the main cause for combined blindness and deafness. It should be noted that the therapy has been granted fast track designation by the FDA.Evercore’s Joshua Schimmer is impressed with Sepofarsen’s results so far. The 5-star analyst explained, “We saw 12 month data help confirm the durability of treatment effect in the pivotal Phase 2/3 dose through 12 months. The strong responses were maintained across visual acuity, mobility and sensitivity endpoints. The Phase 1/2 results bode well for the Phase 2/3 and may help validate the other pipeline opportunities… We remain encouraged by the LCA10 12-month data and see read-through to other larger indications.”Therefore, Schimmer reiterated an Outperform rating on PRQR, along with a price target of $34. Should the target be met, investors stand to pocket a massive 323% 12-month gain. (To watch Schimmer’s track record, click here)The Street is equally confident in the rare disease fighter’s ability to soar over the next 12 months. A Strong Buy analyst consensus is formed of Buy ratings only, 5 to be precise. The average price target is $28.80 and indicates possible upside of 258%. (See ProQR stock analysis on TipRanks) Cellectis SA (CLLS)French gene-editing pioneer Cellectis has been turning heads recently with its novel approach to cancer treatment.The company has developed a gene-editing technology called TALEN and has been making deals with fellow companies to advance effective cancer therapies. It recently announced a deal with Iovance Biotherapeutics, to which Cellectis has granted exclusive global rights for the use of its gene-editing technology to edit a series of cancer treatments known as tumor-infiltrating lymphocytes, or TILs. Cellectis, in return, will receive royalties on sales of gene-edited TILs from Iovance along with development and sales milestone payments.Cellectis also has deals with French pharmaceutical company Servier. The two are collaborating on five targets, including UCART19, a cellular therapy candidate against lymphoid malignancies progressing through trials. Additionally, Cellectis has a partnership with cancer-focused biotech Allogene. The company has been granted a license from Cellectis for the development of ALLO-501, a similar treatment to UCART19 but manufactured using a different process. Phase 1 has started in relapsed/refractory non-Hodgkin lymphoma.Nomura’s Christopher Marai believes Cellectis remains “grossly undervalued vs. peers.” The 4-star analyst noted, “We estimate that even a modest 8x on $80 million in potential royalties (estimate 8% royalties on sales, and up to $185 million in milestones per target) is justified by ALLO’s valuation (that incorporates mostly ALLO-501, in our view). This suggests that UCART19 alone should be worth $640 million in enterprise value to CLLS, or $15/share alone. This does not consider the 69.1% stake in Calyxt (~$125 million enterprise value).”What does this mean, then? It means Marai is bullish on CLLS. The analyst reiterated a Buy rating on Cellectis alongside a price target of $73. Should the figure be met, gains of 300% could be in the cards over the next year. (To watch Marai’s track record, click here)And what does the rest of the Street reckon 2020 has in store for the gene-editing company? Upside is the answer – Cellectis’ Strong Buy consensus rating breaks down into 4 Buys and 1 Hold. The average price target is $42.75 and implies a possible 12-month rise of 134%. (See Cellectis stock analysis on TipRanks) Kura Oncology (KURA)Kura is another biotech fighting the good fight against cancer. The company is focused on treatments for solid tumors and blood cancers, with several candidates in the pipeline.All eyes, though, are currently on the company’s lead candidate, Tipifarnib, an oral nonpeptidomimetic farnesyl transferase inhibitor. It can be used in both solid tumors and hematologic malignancies such as acute myeloid leukemia. The drug is presently in various stages of development for the treatment of several indications, and was recently granted fast track designation by the FDA for the treatment of patients with HRAS-mutant head and neck squamous cell carcinoma (HNSCC).Last month, Kura presented data from its ongoing Phase 2 trial for tipifarnib in peripheral T-cell lymphoma (PTCL) at the American Society of Hematology (ASH). Deutsche Bank’s Konstantinos Aprilakis came away impressed. The analyst stated, “We see the impressive clinical efficacy presented for tipifarnib in AITL at ASH as well as the positive regulatory guidance, especially the low bars for success in the pivotal trial, as encouraging for the company and supportive of an accelerated path forward for tipifarnib in the setting of blood cancers.”To this end, Aprilakis left a Buy rating and price target of $28 unchanged, implying upside potential of a handsome 119%. (To watch Aprilakis’ track record, click here)Other members of the Street are with the Deutsche analyst on Kura. 4 Buy recommendations from analysts tracked over the last three months add up to a Strong Buy consensus rating. The average price target comes in at $29.50 and indicates potential upside of 131%. (See Kura stock analysis on TipRanks)

ProQR (PRQR) doses the first patient in the phase I/II Aurora study on QR-1123, which is being evaluated for treating patients with autosomal dominant retinitis pigmentosa.

ProQR (PRQR) delivered earnings and revenue surprises of 23.91% and -100.00%, respectively, for the quarter ended March 2020. Do the numbers hold clues to what lies ahead for the stock?

My Retina Tracker Program is the highest volume IRD genetic testing program in the U.S. LEIDEN, Netherlands and CAMBRIDGE, Mass, Feb. 06, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, announced today its participation in the Foundation Fighting Blindness My Retina Tracker Program, a collaborative, open access program run by Blueprint Genetics and InformedDNA providing no-cost genetic testing and genetic counseling for individuals with a clinical diagnosis of an inherited retinal disease (IRD) such as Leber’s congenital amaurosis (LCA) and Usher syndrome, amongst others.

ProQR Therapeutics N.V. (PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced the first patient dosed in the Phase 1/2 Aurora clinical trial of QR-1123 in patients with autosomal dominant retinitis pigmentosa (adRP). “From previous clinical trials we have seen that RNA therapies can be a promising approach for patients with inherited retinal diseases and there is a strong need for novel approaches in diseases that currently have no treatments,” said David G. Birch, Ph.D., Principal Investigator of Aurora and Scientific Director of the Retina Foundation of the Southwest in Dallas, Texas.

Daniel de Boer has been the CEO of ProQR Therapeutics N.V. (NASDAQ:PRQR) since 2012. This report will, first, examine...

Before putting in our own effort and resources into finding a good investment, we can quickly utilize hedge fund expertise to give us a quick glimpse of whether that stock could make for a good addition to our portfolios. The odds are not exactly stacked in investors' favor when it comes to beating the market, […]

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) --  March 31, 2020 -- ProQR Therapeutics N.V. (PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. The Company is also providing an update on business operations in relation to the COVID-19 pandemic.

Editas (EDIT) and Allergan are co-developing a CRISPR-based candidate, AGN-151587, for treating Leber congenital amaurosis 10 (LCA10), an inherited form of blindness.

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that Company management will present and discuss findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa on March 31, 2020. Stellar, or PQ-421a-001, is a first-in-human study of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene and is conducted at expert sites in North America and Europe. QR-421a is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

Leiden, Netherlands & Cambridge, Mass., May 25, 2020 -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation.